By Matthew Herper, Forbes Staff Shares in , already the best-performing biotech stock of the past 52 weeks, rose 13% in pre-market trading after the company announced that it plans to submit its muscular dystrophy drug, eteplirsen, to the Food and Drug Administration in the first half of 2014. …read more
Source: FULL ARTICLE at Forbes Latest
What do you think about this?
http://www.cnn.com/2013/07/11/health/gay-blood-drive/index.html?hpt=hp_bn13
— Activists are organizing the first national gay blood drive Friday in an effort to combat the Food and Drug Administration’s ban on blood donations from gay and bisexual men.
The FDA bans donations from men who have had sex with other men since 1977, saying there is an increased risk of exposure to and transmission of infectious diseases — including HIV — in male-to-male sexual encounters.
“FDA uses multiple layers of safeguards in its approach to ensuring blood safety,” the government agency’s website says. The FDA screens all potential blood donors based on risk factors and signs of infections.
Source: Worthy Christian Forums
By Sean Williams, The Motley Fool
Filed under: Investing
Last week we focused on five can’t-miss earnings reports. This week, we’re going to turn the tables back to the Food and Drug Administration and focus on a PDUFA decision and an FDA panel review slated for this week.
Veloci-Raptor time?
First up is Raptor Pharmaceuticals with Procysbi (previously known as RP-103), its oral delayed and extended-release medication to treat nephropathic cystinosis. In trials, Procysbi proved to be non-inferior to the only other FDA-approved treatment for nephropathic cystinosis, known as Cystagon from Mylan .
As my Foolish colleague Keith Speights has already pointed out, both Procysbi and Cystagon are two different forms of cysteamine bitartate. However, Cystagon comes with the need to be taken four times daily (meaning sleep schedules can and will get interrupted) and has a slew of other potential side effects. Procysbi, on the other hand, needs only to be taken twice daily and has milder side effects.
From an ease-of-use standpoint, the drug looks like a slam-dunk for an approval, but the FDA will have to be able to determine that the side effects are diminutive compared to the benefits Procysbi can deliver. If Raptor can get Procysbi approved it would give the company its first revenue generating drug, as well as peak sales potential in the U.S. of $60 million according to JMP Securities. This PDUFA decisions has already been pushed back 90 days to review additional data, so this will certainly be a name to watch on Tuesday.
Will AVEO be on target?
The other FDA decision that should have the attention of biotech investors this week is AVEO Pharmaceuticals‘ Tivozanib, which is set to go before the FDA‘s review panel on Thursday.
Tivozanib is an oral, once-daily treatment for metastatic renal cell carcinoma that works by blocking three VEGR-receptors that are critical for blood vessel growth development for solid renal cell tumors, and is being targeted as a first-line treatment. In trials, AVEO and its partner Astellas Pharma went head-to-head against Onyx Pharmaceuticals‘ Nexavar and delivered good, but not groundbreaking, results. In its TIVO-1 study, released last year, Tivozanib met its primary progress-free survival endpoint and delivered a median PFS of 11.9 months compared with the median PFS of just 9.1 months for Nexavar. The difference was even more pronounced in treatment-naive patients where the PFS difference grew to 12.7 months as compared to 9.1 months for Nexavar. However, overall survival rates for AVEO‘s drug actually were slightly lower than Nexavar over the course of the study.
Regardless of what the FDA panel has to say about Tivozanib, it could be an uphill struggle given that seven drugs are currently approved by the FDA to treat renal cell carcinoma. In addition to the aforementioned Nexavar, Pfizer‘s Sutent is a lead drug in metastatic renal cell carcinoma, delivering $1.24 billion in sales — a 9% year-over-year increase — in 2012.Even if Tivozanib makes it through the gauntlet,
Source: FULL ARTICLE at DailyFinance
By Dan Newman, The Motley Fool
Filed under: Investing
Since 1981, 25 million people have died from HIV/AIDS. An estimated 1.7 million died in 2011 due to AIDS-related causes, with another 2.5 million newly infected with HIV. Beyond the human cost, the U.S. government spent $28 billion fighting HIV in 2012, and some African countries lose 1% in GDP growth each year due to the disease. It’s difficult to grasp these numbers, but it’s a problem crying out for several solutions.
One such solution is offered by Female Health Company : FC2, the only female condom approved by the Food and Drug Administration and the World Health Organization.
Social opportunity
The Bill and Melinda Gates Foundation recently called for proposals for its Grand Challenges in Global Health grant program. One of the topics addressed this year is “reinventing the condom.” As the blog post describes the issue:
It may seem obvious, but the success and impact of any public health tool hinges on that tool being used consistently and correctly by those who need it. Vaccines sitting on shelves don’t prevent disease. New tuberculosis drug regimens won’t help if patients stop taking them halfway through the necessary days. Likewise, the potential value of condoms is limited by inconsistent use.
The FC2 condom is a superb public health tool that allows women to initiate protection against sexually transmitted diseases. As to making sure it is used consistently and correctly, Female Health has a variety of initiatives. In South Africa, Female Health has distributed 20 million condoms and trained 10,000 health providers. The company also runs a website with product information and training, it will be investing $14 million over the next six years in HIV/AIDS and reproductive education with global agencies, and it has set a goal of reaching 120 million more women in the poorest countries by 2020.
Profitable opportunity
Some may guess that doing business with developing countries wouldn’t allow for a very profitable business. However, when South African companies like Sasol — which estimated 18% of its workforce carried HIV in 2007 — must dedicate departmental budgets to HIV/AIDS, there are plenty of opportunities for Female Health to cover costs and earn a return. A healthier workforce for Sasol would simply cost less for the company, and Female Health can help companies like Sasol achieve a healthier workforce.
Closer to home, at least 3% of Washington, D.C., residents carry HIV/AIDS. Working with Johns Hopkins, a study found:
…a public-private partnership to provide and promote FC2 female condoms, prevented enough HIV infections in the first year alone to save over $8 million in avoided future medical care costs (over and above the cost of approximately $445,000 for the program). This means that for every dollar spent on the program, there was a cost savings of nearly $20.
FHCO Revenue TTM data by YCharts.
Risky opportunity
Female Health‘s major customers are from the public sector — like the United Nations Population Fund and USAID — which the company warns can make for lumpy revenue due to
From: http://www.dailyfinance.com/2013/04/18/an-investment-opportunity-for-a-healthy-world/
By Business Wirevia The Motley Fool
Filed under: Investing
Seattle Genetics to Host Conference Call and Webcast Discussion of First Quarter 2013 Financial Results on May 7, 2013
BOTHELL, Wash.–(BUSINESS WIRE)– Seattle Genetics, Inc. (NAS: SGEN) announced today that it will report its first quarter 2013 financial results on Tuesday, May 7, 2013, after the close of financial markets. Following the announcement, company management will host a conference call and webcast discussion of the results and provide a general corporate update. Access to the event can be obtained as follows:
LIVE access on Tuesday, May 7, 2013
1:30 p.m. Pacific Time / 4:30 p.m. Eastern Time
REPLAY access
About Seattle Genetics
Seattle Genetics is a biotechnology company focused on the development and commercialization of monoclonal antibody-based therapies for the treatment of cancer. The company’s lead program, ADCETRIS® (brentuximab vedotin), received accelerated approval from the U.S. Food and Drug Administration in August 2011 and approval with conditions from Health Canada in February 2013 for two indications. In addition, under a collaboration with Millennium: The Takeda Oncology Company, ADCETRIS received conditional marketing authorization from the European Commission in October 2012. Seattle Genetics also has four other clinical-stage ADC programs: SGN-75, ASG-5ME, ASG-22ME and SGN-CD19A. Seattle Genetics has collaborations for its ADC technology with a number of leading biotechnology and pharmaceutical companies, including Abbott, Agensys (an affiliate of Astellas), Bayer, Celldex Therapeutics, Daiichi Sankyo, Genentech, GlaxoSmithKline, Millennium, Pfizer and Progenics, as well as ADC co-development agreements with Agensys and Genmab. More information can be found at www.seattlegenetics.com.
By Business Wirevia The Motley Fool
Filed under: Investing
Baxter Achieves Its First Quarter Expectations and Confirms 2013 Full-Year Guidance
DEERFIELD, Ill.–(BUSINESS WIRE)– Baxter International Inc. (NYS: BAX) today reported first quarter financial results in line with the company’s previously issued guidance and confirmed its full-year 2013 financial outlook.
For the first quarter, Baxter reported net income of $552 million and earnings per diluted share of $1.00, compared to net income of $588 million and earnings per diluted share of $1.04 in the same period last year. First quarter 2013 results include after-tax special items totaling $29 million (or $0.05 per diluted share) primarily for deal-related costs associated with Baxter’s planned acquisition of Gambro AB, a global medical technology company focused on developing, manufacturing and supplying dialysis products and therapies for patients with acute or chronic kidney disease. First quarter 2012 results included a net after-tax benefit from special items totaling $19 million of income (or $0.03 per diluted share) for payments and adjustments pertaining to business development transactions.
On an adjusted basis, excluding special items in both periods, Baxter’s first quarter net income of $581 million increased 2 percent from $569 million reported in 2012. Adjusted earnings per diluted share of $1.05 rose 4 percent from $1.01 per diluted share last year, in line with the company’s previously issued earnings guidance of $1.03 to $1.05 per diluted share.
Worldwide sales of $3.45 billion increased 2 percent compared to $3.39 billion reported in the first quarter of 2012. Sales within the United States increased 1 percent, totaling $1.48 billion, while international sales of $1.97 billion increased 2 percent. The impact of foreign currency on sales growth in the quarter was immaterial.
By business, BioScience revenues of $1.53 billion rose 5 percent, driven primarily by strong demand for ADVATE [Antihemophilic Factor (Recombinant), Plasma/Albumin-Free Method] for the treatment of hemophilia, as well as certain plasma-based therapeutics and vaccines. Medical Products sales of $1.92 billion were comparable to the prior-year period as growth for intravenous therapies was more than offset by lower sales in the company’s pharma-partnering business.
Baxter continued to make investments to support geographic expansion initiatives and new product introductions, and continued to advance a number of programs that improve the quality of care and address key, high-potential areas of unmet medical need. Recent achievements include:
From: http://www.dailyfinance.com/2013/04/18/baxter-achieves-its-first-quarter-expectations-and/
A new reformulated form of Oxycontin (a highly addictive oxycodone hydrochloride) has received a unique labeling update by the U.S. Food and Drug Administration regarding its abuse potential. People most commonly abuse Oxycontin by injecting or snorting it. However, with Purdue Pharma L.P.’s new reformulated version, abuse is much harder. OxyContin abuse is a huge problem in the U.S…
By The Huffington Post News Editors
By Toni Clarke
WASHINGTON, April 16 (Reuters) – The head of the U.S. Food and Drug Administration conceded on Tuesday that the agency could have been more aggressive in its oversight of the compounding pharmacy at the center of a deadly meningitis outbreak.
Testifying at a contentious congressional hearing, FDA Commissioner Margaret Hamburg said a confusing legal landscape, combined with resistance from compounding pharmacies, had hampered her agency’s ability to act on a myriad of complaints against the New England Compounding Center and its sister company, Ameridose LLC.
“I wish we had acted earlier,” Hamburg told a House Energy and Commerce subcommittee looking into the outbreak, which has killed 53 people and sickened more than 700.
The hearing was the second held by the committee to determine whether the FDA could have prevented the outbreak and whether it needs greater powers to regulate pharmacies that compound drugs tailored for specific patients. In the past decade, some have come to operate more like traditional pharmaceutical manufacturers, but without the same oversight.
Hamburg said the current legal framework “does not provide FDA with the tools needed to identify and adequately regulate these pharmacies to prevent product contamination.” She asked Congress to allow the FDA to require compounding pharmacies to register with the agency “so we know who they are and what they do.”
Hamburg also would like to ensure a consistent set of safety regulations that would require compounding pharmacies to report any problems associated with their drugs.
“We are hopeful that the Senate will come out with a legislative proposal soon,” said Steven Immergut, an FDA spokesman.
Drawing on roughly 30,000 pages of documents turned over by the FDA, committee members honed in on the agency’s decision, in 2011, to stop initiating inspections of compounding pharmacies until it had finalized new guidance designed to clarify how it would regulate the industry in the face of a complex set of legal decisions dating back years.
The FDA has not made the documents public, saying they are
By Matthew Herper Sarepta Therapeutics has been one of the most exciting — and, from a stock perspective, best-performing, biotechnology companies this year. One big question: would the limited data Sarepta has so far collected from a very small clinical trial of its drug eteplirsen, a treatment for Duchenne muscular dystrophy that is caused by a very specific type of mutation, be enough to warrant filing with the Food and Drug Administration and maybe even approval?
By Matthew Herper Over the past 10 years, 278 new drugs have been approved by the Food and Drug Administration, according to data from the InnoThink Center for Research in Biomedical Innovation. One hundred-and-seventy-five of them, or 63% of those approvals, came from 41 companies that had at least two new drugs approved during that time period. (Specifically, we’re looking from the beginning of 2003 until now.) Below are all of those 41 companies.
By Sean Williams, The Motley Fool
Filed under: Investing
Earnings season is getting ready to rev into high gear, but earnings are unlikely to be the talk of the sector in the days to come, with three big Food and Drug Administration actions expected in the coming week.
No headaches for Allergan shareholders
Up first is the PDUFA decision on Allergan‘s inhaled migraine medication Levadex on Monday. MAP Pharmaceuticals received a complete response letter for Levadex in March 2012 because of manufacturing deficiencies and its delivery device. Levadex wasn’t denied, though, based on safety or efficacy. This was a clean cue to investors that Levadex’s approval seemed like a sure thing barring it worked hand-in-hand with the FDA to meet its demands. With Allergan taking that cue as a reason to buy MAP earlier this year for $958 million, Monday represents decision day as to whether the company has made adequate fixes to its delivery device and manufacturing process to satisfy the FDA.
Levadex’s approval or rejection could also mean a good or bad day for Nektar Therapeutics , which looks to gain from royalty rights based on its contributions to Levadex’s development. While impossible to predict, I’m going to go out on a limb and project an approval for Allergan.
A revolutionary COPD treatment?
On Wednesday, chronic obstructive pulmonary disease, or COPD, treatment collaborators GlaxoSmithKline and Theravance are set to go before the FDA‘s panel with their combo Breo Ellipta. This revolutionary drug combines Theravance’s long-acting beta-2 agonists with Glaxo’s long-acting muscarinic antagonists into a dry powder inhaler to provide long-term COPD relief.
Studies of Breo Ellipta didn’t show statistically significant effects at all doses examined (clinical trials are a bit of trial and error on dosing), but they all showed a measurable improvement in lung function, although not all increases were dubbed significant. In addition, the adverse event profile of Breo Ellipta was similar to the current standard of treatment.
As for Wednesday, I believe the patient pool should be enough to satisfy the FDA panel, but I’m concerned that it may focus on some of its non-statistically significant trials as a sticking point. Overall, I’m leaning toward a positive review from the FDA‘s panel, but we’ll find out more on Wednesday.
Is this the End-o for Aveed?
Finally, on Thursday we have an FDA panel meeting for Endo Health Solutions‘ Aveed, with is an extended-release male hypogonadism treatment.
The drug itself was acquired when Endo purchased Indevus Pharmaceuticals in early 2009 and is on its second go-around after receiving a complete response letter in its first attempt to gain approval. That CRL spelled out the FDA‘s concerns regarding very rare, but serious, adverse side effects, which included post-injection anaphylaxis and pulmonary oil microembolism. The FDA also noted that Endo’s risk evaluation and mitigation strategy was insufficient.
It’s been more than three years since that initial CRL, so this should definitely be an interesting FDA panel meeting to say the
From: http://www.dailyfinance.com/2013/04/14/3-cant-miss-fda-actions-this-week/
By Sean Williams, The Motley Fool
Filed under: Investing
It was a good two months late, but yesterday both political parties and the American people got to read the fine print of President Barack Obama‘s proposed 2014 federal budget.
As you might expect, quite a few Democrats supported the main points of the budget, and House Republicans in almost every respect shunned the president’s outlined measures, which included higher taxes for upper-income earners and some spending cuts as well. Ultimately, there wasn’t much new and the bipartisan bickering that ensued has become a norm on Wall Street for the past couple of years.
82 billion reasons to quit
However, what did stick out like a sore thumb was Obama‘s budget proposal that entailed raising the federal tax on cigarettes from $1.01 per pack to $1.95 – a $0.94 increase! According to calculations associated with the president’s budget proposal, the increase would raise $78 billion over the next decade, which would be enough to fund a universal preschool program for children.
Source: White House.
In addition, it’s estimated that $1 billion in long-term health care costs would be saved from a reduction in smoking caused by the increase in prices, and $3 billion would be added to the economy thanks to a healthier labor force. Added together, Obama‘s plan to smoke the tobacco industry — the irony here is that the president was once a smoker himself — could result in an $82 billion favorable swing over the next decade.
While the magnitude of the tax provision astounded me, I’m not actually surprised to see a cigarette tax hike included in the president’s budget proposal with the implementation of the Patient Protection and Affordable Care Act, known as Obamacare, right around the corner. The president understands the costs associated with implementing a wave of health reform, and also understands that minimizing as many long-term health problems as possible (i.e., getting young people to stop smoking) will help his health care reform bill achieve success.
No ifs, ands, or butts about it!
Big tobacco companies, on the other hand, definitely didn’t take kindly to the president’s proposal, which represents the latest threat in a series of events meant to increase the public’s awareness of the dangers of smoking.
Last year, the Centers for Disease Control and Prevention took aim at the tobacco industry with a $54 million, three-month long marketing blitz advertising the dangers of cigarette smoking, while the Food and Drug Administration exerted its force on the industry by requiring tobacco producers to disclose the quantities of 20 known harmful chemicals found in cigarettes.
Before even the CDC and FDA got involved, individual cities and locales, such as New York, got involved by banning cigarette smoking inside restaurants and in largely public places like plazas and beaches.
The weight of these measures on domestic tobacco companies is really going to start taking its toll. Even if Obama‘s budget proposal fails to pass through Congress — and every political think
From: http://www.dailyfinance.com/2013/04/11/president-obama-unveils-a-plan-to-smoke-big-tobacc/
By Brian Orelli, The Motley Fool
Filed under: Investing
There goes the short-term bear thesis on ACADIA Pharmaceuticals .
I had recommended staying away from the company for now because it looked too risky with a long wait before the company could complete its second phase 3 trial for pimavanserin in patients with Parkinson’s disease psychosis.
But after talking to the Food and Drug Administration, ACADIA said today that the agency has agreed that the current data is “sufficient to support the filing of a New Drug Application.” That doesn’t guarantee the FDA is going to approve the drug, but it’s certainly a positive sign.
The FDA generally requires at least two successful phase 3 trials, but will accept a single trial in certain instances when there’s an unmet need. There aren’t any drugs approved to treat Parkinson’s disease psychosis, although atypical antipsychotics such as Johnson & Johnson‘s Risperdal, Eli Lilly‘s Zyprexa, Bristol-Myers Squibb‘s Abilify, and Pfizer‘s Geodon are used off label to treat Parkinson’s patients experiencing psychotic symptoms, which affects up to 60% of Parkinson’s patients.
Shares of ACADIA are up more than 50% as I write this. That’s well deserved.
Not having to do the study should speed things up a little — ACADIA hadn’t even started the second phase 3 trial — but not by the full length of the trial. There are still drug-drug interaction studies and chemistry and manufacturing tests that need to be completed; the company doesn’t expect to file the marketing application until near the end of next year.
So, why are shares up so much? Not having to run a confirmatory trial reduces the risk substantially.
Pimavanserin produced solid data in its most recent trial. Patients taking the drug saw their SAPS-PD score, a measurement of hallucinations and delusions, drop by 5.79 points while scores for placebo patients only dropped 2.73 points. The difference was statistically significant.
But this wasn’t the first trial testing pimavanserin, or even the second. ACADIA ran two previous trials, which both saw patients in the placebo group improve substantially based on the assessment scale. For the latest successful trial, the company modified the scale — that’s the PD in SAPS-PD — to include the nine items most relevant to patients with psychosis associated with Parkinson’s disease.
The modification clearly worked, but there are no guarantees that it could be repeated. Patients entering the confirmatory trial would know that the drug had succeeded, which could boost the scores of the placebo patients.
So, is ACADIA a buy now? With the risk removed, it’s more appealing, but you have to be willing to wait. We’re still a year and a half away from filing the application, and then there’s another eight to 12 months before pimavanserin will be approved.
With a market cap of $950 million, there’s some room to run once the drug hits the market in 2015. Pimavanserin shouldn’t have too much trouble taking away sales from Risperdal, Zyprexa, Abilify, Geodon, and the like since it’ll be approved
From: http://www.dailyfinance.com/2013/04/11/fda-throws-acadia-a-juicy-bone/
They look and feel like real cigarettes, but they’re much more than that. Doubling in market share annually, electronic cigarettes are taking off. Introduced to the U.S. market in 2007, e-cigarettes give smokers the freedom to smoke whenever and wherever they want, tobacco-free, while simultaneously quitting their traditional smoking habits. But there are still a lot of question surrounding the boom of the e-cig. In the U.S., e-cigs aren’t subject to tobacco laws and the Food and Drug Administration has yet to officially speak out against them. E-cig brands seem to be popping up everywhere. Presented as healthier, cheaper, and more environmentally-friendly than cigarettes, smokers are using them to break their habit and celebrities are endorsing them–but this new high-tech smoking trend presents a variety of aspects worth a second look. Components Of E-Cigs It looks like a cigarette, smokes like a cigarette, but doesn’t damage or smell like one. This product is a tobacco-free simulation of the cigarette experience. It’s actually an electronic inhaler. The flame is simulated, along with the vapor smoke, and plastic ash tip. The whole experience is “fake,” but satisfying. E-cigarette smokers inhale and exhale a liquid nicotine vapor heated by a battery; the amount of nicotine is chosen by the user.
By Sean Williams, The Motley Fool
Filed under: Investing
Although we don’t believe in timing the market or panicking over market movements, we do like to keep an eye on big changes — just in case they’re material to our investing thesis.
What: Shares of ACADIA Pharmaceuticals , a clinical-stage biopharmaceutical company, skyrocketed as much as 54% after it announced that it was filing an accelerated new drug application for Pimavanserin after discussions with the Food and Drug Administration.
So what: Pimavanserin, an experimental anti-psychosis drug for people with Parkinson’s disease, breezed through its late-stage clinical trial, meeting the primary endpoint of “highly significant antipsychotic activity,” and also meeting the secondary endpoint of improved motoric tolerability. ACADIA had been planning to run a confirmatory phase 3 trial, which it planned to begin enrolling patients in this quarter. However, the data thus far, and the discussion between the FDA and ACADIA, warranted an early drug submission.
Now what: I’d say this is definitely a step in the right direction toward getting Pimavanserin approved. But, let’s also keep in mind that what the FDA does initially and what its panel or final ruling may indicate can occasionally be two different things. Another factor to consider is that most Wall Street peak sales estimates for Pimavanserin are around $300 million within the U.S. Acadia’s valuation is pushing $950 million following today’s pop, meaning it’s valued at more than three times peak sales. To me, that seems a bit lofty for its first potential FDA-approved drug.
Craving more input? Start by adding ACADIA Pharmaceuticals to your free and personalized watchlist so you can keep up on the latest news with the company.
While you can certainly make huge gains in biotechs like ACADIA, the best investing approach is to choose great companies and stick with them for the long term. The Motley Fool’s free report “3 Stocks That Will Help You Retire Rich” names stocks that could help you build long-term wealth and retire well, along with some winning wealth-building strategies that every investor should be aware of. Click here now to keep reading.
The article ACADIA Pharmaceuticals Skyrockets on Accelerated NDA Filing originally appeared on Fool.com.
Fool contributor Sean Williams has no material interest in any companies mentioned in this article. You can follow him on CAPS under the screen name TMFUltraLong, track every pick he makes under the screen name TrackUltraLong, and check him out on Twitter, where he goes by the handle @TMFUltraLong.
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From: http://www.dailyfinance.com/2013/04/11/acadia-pharmaceuticals-skyrockets-on-accelerated-n/
By Sean Williams, The Motley Fool
Filed under: Investing
The process of developing a drug from start to finish is incredibly arduous, usually involves around $1 billion in costs, and can take upwards of a decade to complete. To boot, a vast majority of drug hopefuls are destined to fail. That’s why yesterday’s news from Pfizer and the Food and Drug Administration should get breast cancer sufferers’ blood pumping.
What’s behind the “breakthrough” designation
The FDA yesterday gave Pfizer’s Palbociclib the extremely rare and relatively new “breakthrough therapy” designation based on its phenomenal mid-stage trial results and the life-threatening nature of some of the cancers it could eventually treat.
The “breakthrough therapy” designation is about three months old — it was a designation added to the Food and Drug Administration Safety and Innovation Act last year — and is targeted at expediting experimental drugs through the development and review process. According to FDASIA, a breakthrough therapy is one that, “treat a serious or life-threatening condition, and preliminary clinical evidence indicates that the drug [or combination of drugs] may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.”
Very few breakthrough therapies have been approved up to this point — then again, we’re only working with about 100 days for the Food and Drug Administration to have assigned such a designation. Vertex Pharmaceuticals‘ received the first two “breakthrough therapy” designations from the FDA for two cystic fibrosis treatments, of which both involve the use of the already FDA-approved Kalydeco. The more intriguing “breakthrough therapy” designation is Pharmacyclics‘ Ibrutinib for the treatment of two rare blood cancers, which analysts feel could have a peak sales value of $5 billion if approved.
The awe-inspiring numbers behind the designation
The potential for Vertex’s cystic fibrosis dynamic duo and Pharmacyclics’ Ibrutinib in clinical trials so far cannot be refuted, but I don’t believe they hold a candle to the ridiculously strong clinical data that Pfizer’s combination of Palbociclib and Novartis‘ Femara brought to the table in its mid-stage metastatic breast cancer trial.
The decision to assign the “breakthrough therapy” designation had to do with the end results, which showed Femara by itself providing trial patients with 7.5 months of progression-free survival, or PFS, while the combination of Palbociclib and Femara more than tripled that level to 26.1 months of PFS. I read that and my jaw hit the floor! That is the precise definition of a breakthrough drug that, if safe in trials, should be ushered through the development and review process with the utmost urgency.
Palbociclib itself targets ER+, HER2-positive breast cancer (the most common form of breast cancer) and works by inhibiting two cyclin dependent kinases, or CDKs, 4 and 6. These CDKs are essential for cell replication and their suppression has been demonstrated to interfere with tumor cell progression in advanced stages of the disease. Analysts at both Leerink Swan and JPMorgan estimate that, if approved for multiple indications, Palbociclib could have sales
From: http://www.dailyfinance.com/2013/04/11/a-breakthrough-may-be-on-the-way-for-breast-cancer/
By Zina Moukheiber, Contributor A study published today in Nature Clinical Pharmacology and Therapeutics showcases the potential power of sophisticated data analytics when applied to electronic health records on a large scale. By predicting harmful drug side effects two years before an alert from the Food and Drug Administration, the technology can complement the FDA’s Adverse Event Reporting System, which collects information from doctors, pharmacists, and patients once a drug is on the market. Less than three years ago, bioinformaticians at Stanford University set out to capture information buried, not just in insurance claims, lab tests or medical codes, but in the rich narrative of 10 million clinical notes gleaned from 1.8 million patients on Epic Systems electronic health records. “Ninety percent of patient data comes from clinical notes,” says senior author Nigam Shah, who teaches biomedical informatics at Stanford. Along a binary matrix, Shah and his colleagues plotted de-identified patients, diagnoses, drugs and outcomes, among other variables. Patterns emerged detecting which patients, for example, with rheumatoid arthritis took Vioxx, and subsequently suffered a heart attack, as well different variations of the same variables. Drug dosages were not taken into account. Shah and his Stanford colleagues tested their model on nine drugs that were slapped with strong warnings after deadly side effects emerged, including Vioxx and Avandia. Their algorithms were able to detect adverse effects in six of them, two years before the FDA’s alert. (Stanford had 15 years of patient data). Shah says they didn’t have enough patients to get statistically significant information on three drugs, but that will change as more patient data gets stored in electronic health records.
Source: FULL ARTICLE at Forbes Latest
By Brian Orelli, The Motley Fool
Filed under: Investing
Gilead Sciences moved one step closer to an all-oral cure for hepatitis C on Monday when the company submitted its application to the Food and Drug Administration to market sofosbuvir for the viral infection.
The current regimen for patients with hepatitis C involves taking one of two oral drugs — either Merck‘s Victrelis or Vertex Pharmaceuticals‘ Incivek — plus an oral generic called ribavirin and a branded a pegylated interferon drug, either Merck’s PegIntron or Roche‘s Pegasys. Pegylated interferons have to be injected, which patients generally don’t like, and more importantly, they also cause uncomfortable side effects, including flu-like symptoms. Considering they have to be taken for six months or more, it’s understandable why patients don’t like them.
Short-term subdued
Sofosbuvir passed four phase 3 trials. It seems very likely that the FDA will approve the drug in eight to 12 months depending on whether the drug is given a priority review.
But for most hepatitis C patients, sofosbuvir isn’t a cure-all by itself. When the drug is combined with ribavirin, it does a pretty good job at curing patients with genotype 2 and 3, but the most common genotype in the U.S. is genotype 1. Patients with genotype 1, along with genotype 4, 5, and 6, will still have to take PegIntron or Pegasys and ribavirin if sofosbuvir is approved.
While some patients will do just that, many will wait. Hepatitis C is a slow -cting disease. Eventually the virus starts to damage the liver, including potentially causing liver cancer, but it takes a long time to get that far. Most patients can wait for a better treatment.
They’re already doing that. Sales of Incivek and Victrelis peaked shortly not long after their launches as patients wait for better treatments. Sales of sofosbuvir will likely follow the same path. At least initially.
Long-term lucrative
Gilead is testing sofosbuvir in combination with another oral medication, GS-5885, with and without ribavirin in genotype 1 patients. If the phase 3 trials turn out positive, the additional data could be added to the label after it’s approved. Doctors can prescribe it off-label before that, but they’d have to wait for GS-5885 — or a fixed-dose combination pill that contained both medications — to be approved.
How big of a blockbuster sofosbuvir becomes depends on how good the data is compared to other all-oral cocktails being developed by AbbVie , Vertex, Johnson & Johnson , and many others. The cure rates will be important, but as they exceed 90%, safety and the amount of time patients have to take the drug will become more important.
Gilead, for instance, is testing sofosbuvir and GS-5885 with and without ribavirin for eight weeks. Even if it doesn’t beat AbbVie’s all-oral cocktail, which produced a 99% cure rate in one of its phase 2 trials, a shorter time frame might help attract patients.
The other main driver for sales will be how many patients
Source: FULL ARTICLE at DailyFinance
By Brian Orelli, The Motley Fool
Filed under: Investing
If one “Breakthrough Therapy Designation” is good, three must be great.
That is, if we knew what the designation was really worth.
Johnson & Johnson announced on Monday that it had received a third Breakthrough Therapy Designation for the blood cancer drug ibrutinib that it’s developing with Pharmacyclics . The designation covers patients with chronic lymphocytic leukemia or small lymphocytic lymphoma who have a deletion of the short arm of chromosome 17.
In February, the Food and Drug Administration gave the drug the designation for patients with relapsed or refractory mantle cell lymphoma that failed prior therapy and for patients with Waldenstrom’s macroglobulinemia, a type of cancer affecting B cells.
The Breakthrough Therapy Designation was part of the last year’s FDA Safety and Innovation Act, which is supposed to speed up development review time — potentially requiring less data for approval — for drugs that treat serious diseases where there isn’t a treatment or where the product in development is a vast improvement over current options.
But Congress, in its infinite wisdom, left exactly how to implement the benefits up to the FDA. That was probably a good idea in the long run since the FDA knows what drugmakers need, but the agency isn’t particularly quick in establishing rules.
Companies are left making announcements about a designation that they don’t know exactly how they’ll benefit from. “The implications of Breakthrough Therapy Designation cannot be determined at this time,” read the press release by Johnson & Johnson’s subsidiary Janssen Research & Development.
Vertex Pharmaceuticals had the exact same wording — maybe their flack went to the same law school — in the announcement that the FDA had given Breakthrough Therapy Designation to its cystic fibrosis drug Kalydeco as both a monotherapy and in combination with VX-809.
The biotech added that it’s working with the FDA and other regulatory authorities to figure out exactly how it’ll affect the development. It sure doesn’t look like the designation will help reduce the size of the clinical trials required for approval. Vertex subsequently announced that it’s started two 500-patient phase 3 trials testing VX-809 with Kalydeco in cystic fibrosis patients with two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene.
The designation might not have much benefit, but don’t ignore the drugs involved; both Vertex’s combination treatment and ibrutinib have a lot of promise. Just this week, promising data from a clinical trial testing ibrutinib in hart-to-treat chronic lymphocytic leukemia patients was presented at the American Association for Cancer Research annual meeting.
At this point, the Breakthrough Therapy Designation is like wearing a broken luxury watch; it’s a nice status symbol, but it isn’t particularly useful. Some will argue that it says that the FDA thinks the drug has promise, but that should be obvious to most investors anyway. The drugs still have to prove their worth in clinical trials to get approved.
Is bigger really better?
Involved in everything from baby powder to biotech, Johnson & …read more
Source: FULL ARTICLE at DailyFinance
By Max Macaluso, Ph.D., The Motley Fool
Filed under: Investing
Last summer, the Food and Drug Administration approved two new medications for the treatment of obesity. VIVUS‘ Qsymia has already reached the market, while Arena Pharmaceuticals‘ Belviq will be launched by its commercialization partner Eisai once the drug clears DEA scheduling. A third player, Orexigen Therapeutics , is still developing its drug Contrave. The fact that obesity affects more than a third of American adults today has attracted many biotech investors to these stocks, but many may not fully understand how physicians approach treatment for this disease. For instance, are exercise and diet all patients really need to manage their weight?
To help clarify this complex topic, Motley Fool health-care analyst Max Macaluso spoke with Dr. Domenica Rubino, a weight-management expert and representative of The Obesity Society. In the following segment from their discussion, Dr. Rubino discusses the role that drugs play in treating obesity. A transcript follows the video.
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The relevant video segment can be found between 5:20 and 8:17.
Max Macaluso: Let’s pivot the discussion to drugs and treatment now. Arena recently got Belviq approved. It’s not on the market yet. Vivus got Qsymia approved, and Orexigen is still developing Contrave. What role do drugs really play in treatment?
Dr. Domenica Rubino: A fundamental thing to understand is that the physiology of obesity is actually very complicated. Just as I was saying there are multiple causes of obesity, there are multiple factors that protect weight.
What people don’t really understand is that the body is totally focused on holding onto weight, at all costs. People like to say, “Well, you just eat less, move more.” It’s not as simple as that, because when we do lose weight — and countless people who struggle with this lose weight — there are a lot of mechanisms that kick in.
There are endocrine signals coming from the fat cells, muscle cells, the GI tract, all to the brain to say, “Hold onto that weight.”
What happens to that person is they get hungrier, they don’t really want to move, certain foods look much more appealing, certain parts of the brain are lighting up, thinking, “Oh, I want that.”
What else can happen is that people start to lose their sense of portion. The same subject, before weight loss and after weight loss, will not be able to estimate that portion anymore. Your body is in full-court press to regain weight.
That’s where these medications can be really helpful. They augment our toolbox, as physicians, because primarily what we work with is helping people make lifestyle changes, identifying those factors that I was talking about.
Do they have sleep apnea? …read more
Source: FULL ARTICLE at DailyFinance
