Tag Archives: New Drug Application

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FDA Throws ACADIA a Juicy Bone

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By Brian Orelli, The Motley Fool

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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/

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Merck Announces FDA Acceptance of New Drug Application for an Investigational Tablet Formulation of

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By Business Wirevia The Motley Fool

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Merck Announces FDA Acceptance of New Drug Application for an Investigational Tablet Formulation of the Antifungal NOXAFIL ® (posaconazole)

WHITEHOUSE STATION N.J.–(BUSINESS WIRE)– Merck (NYS: MRK) , known as MSD outside the United States and Canada, today announced that its New Drug Application for an investigational, tablet formulation of the company’s antifungal agent, NOXAFIL® (posaconazole), has been accepted for review by the U.S. Food and Drug Administration (FDA).

Merck currently markets NOXAFIL Oral Suspension for prophylaxis of invasive Aspergillus and Candida infections in patients 13 years of age and older who are at high risk of developing these infections due to being severely immunocompromised, such as patients who have received hematopoietic stem cell transplants and have graft-versus-host disease, or patients with cancers of the blood who are experiencing prolonged low white blood cell counts (neutropenia) as a result of chemotherapy.

“Invasive fungal infections are a significant cause of illness and death among severely immunocompromised patients,” said Robin Isaacs, M.D., vice president, infectious disease clinical research, Merck Research Laboratories. “This filing for a tablet formulation of NOXAFIL is an example of Merck’s ongoing commitment to developing new therapy options for patients in the hospital setting.”

Merck is seeking FDA approval of NOXAFIL tablets for once-daily administration (following a twice-a-day loading dose on the first day of therapy). The company has filed a marketing authorization application for NOXAFIL tablets with the European Medicines Agency (EMA) and plans to seek regulatory approval for the tablet formulation in other countries around the world.

Selected safety information about NOXAFIL (posaconazole) Oral Suspension

NOXAFIL is contraindicated in persons with known hypersensitivity to posaconazole, any component of NOXAFIL, or other azole antifungal agents.

NOXAFIL is contraindicated with sirolimus. Concomitant administration of NOXAFIL with sirolimus increases the sirolimus blood concentrations by approximately 9-fold and can result in sirolimus toxicity.

NOXAFIL is contraindicated with the CYP3A4 substrates that prolong the QT interval. Concomitant administration of NOXAFIL with the CYP3A4 substrates pimozide and quinidine may result in increased plasma concentrations of these drugs, leading to QTc prolongation and rare occurrences of torsades de pointes.

NOXAFIL is contraindicated with HMG-CoA reductase inhibitors that are primarily metabolized through CYP3A4 (e.g., atorvastatin, lovastatin, and simvastatin) as increased plasma concentration of these drugs can lead to rhabdomyolysis.

NOXAFIL is contraindicated with ergot alkaloids. NOXAFIL may …read more

Source: FULL ARTICLE at DailyFinance

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3 Speculative Biotech Companies Primed to Pop

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By Keith Speights, The Motley Fool

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They don’t have any products on the market. They have little or no revenue. Earnings? Nada. Many people are betting that they will fail. And yet they could present some of the best investing opportunities on the market today. Here are three speculative biotech companies that appear to be primed to pop in the coming months.

All set to accelerate
With gains of 450% over the last year, one might be inclined to think that Sarepta Therapeutics has done all the popping it’s going to do. I don’t expect another run that large, but Sarepta still has plenty of upside potential.

The biotech company reported fantastic results in October from a phase 2 clinical study of eteplirsen in treating Duchenne muscular distrophy, or DMD. These results were so good that Sarepta is talking with the Food and Drug Administration about the possibility of moving ahead with accelerated approval. If the FDA gives early approval to eteplirsen, Sarepta’s stock will be off to the races again.

It’s far from a sure thing that the FDA will grant accelerated approval, though. To do so requires that the agency accept data from the phase 2 study showing eteplirsen’s increased production of dystrophin as a solid surrogate endpoint in lieu of demonstrating actual clinical efficacy. The FDA could determine that this data isn’t strong enough to warrant accelerated approval.

Even if Sarepta doesn’t win this faster path to market, I expect that eteplirsen will ultimately gain approval regardless. The drug looks to be a game-changer for DMD patients — and should be for Sarepta shareholders also. 

Zooming with Zerenex
Speaking of game-changers, Keryx Biopharmaceuticals could have one for end-stage renal disease, or ESRD, patients. Keryx announced outstanding results from a phase 3 study of Zerenex in January. The drug lowered serum phosphorus levels in ESRD patients significantly and demonstrated a good safety profile to boot.

Keryx shares are up a whopping 150% so far this year but have hovered around $7 per share for the past month. Is the stock poised for more big gains? I think so.

The biotech plans to submit a New Drug Application, or NDA, to the FDA in the second quarter. It will also follow up by mid-year with filing for European regulatory approval. Zerenex seems likely to garner positive decisions on both fronts. I suspect the submissions themselves could serve as mini-catalysts for the stock as investors are reminded about the potential for the drug.

There are some concerns, though. A couple of months ago, IPD Analytics questioned whether Zerenex would be granted New Chemical Entity, or NCE, status by the FDA. NCE status gives a drug five years of exclusivity. My view is that Keryx’s patent protection for Zerenex will be stout enough to protect the drug from generic rivals for quite a while even if NCE status doesn’t come through. 

Ready for a giant leap
MannKind stands closer than ever to taking a giant leap for its shareholders. The company expects …read more

Source: FULL ARTICLE at DailyFinance

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The Big Weight-Drug Wait

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By Keith Speights, The Motley Fool

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“Wait” seems to be the operative word for the obesity drug industry these days. Investors in VIVUS  are still waiting to see if Qsymia can attain the levels of commercial success that they anticipated. Arena Pharmaceuticals  anxiously awaits finalization of scheduling for Belviq by the slow-moving U.S. Drug Enforcement Administration. But the biggest wait of all belongs to Orexigen Therapeutics . Here’s the latest on the waiting game for the third potential weight-loss drug to hit the market — Orexigen’s Contrave.

In a hurry
After the initial New Drug Application, or NDA, for Contrave was not approved by the U.S. Food and Drug Administration, Orexigen hurried to move forward with the cardiovascular study needed to satisfy the FDA. The company launched this additional research, called the Light Study, in June. By early July, Orexigen announced that enrollment was going much faster than initially expected and could wrap up in first quarter of 2013 — taking around half the time originally anticipated.

That outlook actually proved to be pessimistic. Orexigen completed screening for the Light Study by mid-December, enrolling around 9,000 patients to participate in the study and cutting off more than a year from the initial timetable. The next major milestone for the research will be an interim analysis. That analysis can’t occur until 87 or so major adverse cardiovascular events, or MACE, occur with the patients. After the herculean efforts to get the study going so rapidly, Orexigen must now essentially wait for bad things to happen.

In January, the company announced that the resubmission process for the Contrave NDA could be hurried along somewhat. The FDA will allow a summary report from the Light Study interim analysis to be used with the NDA in lieu of a complete report. While the complete clinical study report will be required within 60 days of the resubmission, this decision cuts time out of the process.

How long will the waiting game take? Orexigen says that plans are to submit the NDA again by the end of this year. However, company executives have hedged in recent comments, stating that this time frame could be pushed back to early 2014 if the MACE rate is on the low end of the target 1% to 2% range.

Late to the game?
A key question for investors looking at Orexigen relates to how successful Contrave can be as a late entrant to the obesity drug market. With Qsymia already on the market and Belviq likely to launch in the U.S. any day now, will Contrave be too late to the game? The answer is: “It depends.”

If Arena and VIVUS manage to achieve tremendous success and develop great patient and prescriber loyalty for their drugs, Contrave could face an uphill battle to gain a foothold. VIVUS recently introduced promotions that appear to be designed to attract and hold on to customers in anticipation of near-term competition from Belviq. With the earliest possible commercial launch of Contrave still over a …read more
Source: FULL ARTICLE at DailyFinance

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3 FDA Decisions You Need to Watch in 2013

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By Keith Speights, The Motley Fool

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Sequester or no sequester, the folks at the Food and Drug Administration look to be quite busy in 2013. Their schedules are full with plenty of important decisions that could greatly impact the fates of biotech and pharma companies — and the fortunes of shareholders as well. Here are three FDA decisions that you should watch over the coming months.

1. Will Vascepa be allowed to play limbo?
Much ado has been made about when the FDA will decide on New Chemical Entity, or NCE, status for Amarin‘s cholesterol drug Vascepa and what that decision will be. However, a much more significant FDA decision this year for Amarin will be related to the supplemental New Drug Application, or sNDA, submitted by the company in late February for use of Vascepa in patients with less severe levels of triglycerides.

Vascepa, a highly refined fish oil pill, is currently approved for patients with severe hypertriglyceridemia, which involves triglyceride levels greater than or equal to 500 mg/dL. If the recently submitted sNDA is ultimately approved, patients with greater than or equal to 200 mg/dL triglyceride levels will be able to take Vascepa. This submission is extremely important for Amarin, because the number of patients in this group is around 10 times the size of those with severely high triglyceride levels. 

Amarin should hear from the FDA within the next couple of months about whether the sNDA has been accepted for review. If it is accepted, the final decision on approval for the new indication would be given in late 2013. If Vascepa is allowed to play limbo by lowering the bar for triglyceride levels, Amarin’s shares should skyrocket.

Antares Pharma anxiously awaits an FDA decision in October for Otrexup, a self-injection for the treatment of rheumatoid arthritis. The FDA announced on Feb. 27 that Antares’ NDA had been accepted for review.

Otrexup uses Antares’ Medi-Jet parenteral drug delivery system to allow patients to self-administer methotrexate, or MTX. An estimated 70% of the 1.3 million Americans with rheumatoid arthritis take MTX alone or with another therapy. MTX is available in tablet form or via injection, but better absorption of the drug is obtained through injection. Antares hopes to capture a nice chunk of the market for those patients who currently self-administer with conventional needles or pen injectors.  

Should the FDA approve Otrexup, the prospects for Antares seem to be quite good. I suspect that many patients will like the idea of taking MTX with the easier self-injection approach offered by the Medi-Jet technology. 

3. A Tivo for kidney cancer?
By the end of July, we’ll know if a “Tivo” for kidney cancer will become available. I’m referring to Tivopath, AVEO Pharmaceuticals‘ proposed brand name for its drug tivozanib. The FDA accepted the NDA for tivozanib in November and set a final decision date of July 28 of this year.

Tivozanib targets treatment of an advanced form of kidney cancer that occurs in more than 250,000 …read more
Source: FULL ARTICLE at DailyFinance

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pSivida Reports Updates on ILUVIEN® for Planned Resubmission to FDA and European Launch

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By Business Wirevia The Motley Fool

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pSivida Reports Updates on ILUVIEN® for Planned Resubmission to FDA and European Launch

WATERTOWN, Mass.–(BUSINESS WIRE)– pSivida Corp. (NAS: PSDV) (ASX:PVA), a specialty pharmaceutical company that is a leader in the development of sustained release ophthalmic drug treatments, today announced that its licensee Alimera Sciences, Inc. reported a number of updates with respect to ILUVIEN® for chronic diabetic macular edema (DME).

Alimera announced that it intends to resubmit its New Drug Application for ILUVIEN for DME to the U.S. Food and Drug Administration (FDA) by the end of March 2013. Using data from Alimera’s two previously completed pivotal Phase III clinical trials (FAME™ Study), the resubmission will focus on the safety aspects of ILUVIEN and the population of patients with chronic DME considered insufficiently responsive to available therapies, the same group for which marketing approval for ILUVIEN has been granted in various EU countries, according to Alimera. Approval in the U.S. would entitle pSivida to a $25 million milestone payment from Alimera and 20% of net profits, as defined, from U.S. sales of ILUVIEN by Alimera.

Alimera also announced that shipments of ILUVIEN to the German market are expected to begin in the second quarter of 2013 upon acceptance from the Medicine and Health products Regulatory Agency of the intended commercial batch size, a delay from Alimera’s previous expectation that this would occur in the first quarter of 2013. Alimera further reported that it also expects to begin shipments to the U.K. in the second quarter of 2013 for treatment of privately insured patients.

Alimera reported the submission of a patient access scheme (PAS) for ILUVIEN for DME has been agreed to by the UK’s Department of Health and is now under consideration by NICE for inclusion in its rapid review facility. NICE had previously issued final guidance that ILUVIEN is not a cost-effective treatment for chronic DME considered insufficiently responsive to available therapies. Alimera reported that under the review facility, NICE is expected to assess the impact of the PAS on ILUVIEN‘s cost effectiveness and determine whether an update to the final guidance is warranted.

About pSivida Corp.

pSivida Corp., headquartered in Watertown, MA, develops tiny, sustained release, drug delivery products designed to deliver drugs at a controlled and steady rate for months or years. pSivida is currently focused on treatment of chronic diseases of the back of the eye utilizing its core technology systems, Durasert™ and BioSilicon™. The injectable, sustained release micro-insert ILUVIEN® for the treatment of …read more
Source: FULL ARTICLE at DailyFinance

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2 Sides of Celgene's Apremilast Results

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By Keith Speights, The Motley Fool

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Celgene has good news — and it has bad news. The company announced phase 3 results for apremilast in treating psoriasis at the American Academy of Dermatology meeting this weekend in Miami. Here are both sides of the apremilast news.

Bad news
The phase 3 results were a considerable letdown when compared to phase 2 results. Back in December 2009, Celgene announced phase 2 results of apremilast in treating psoriasis. In that mid-stage trial, 41% of patients taking the drug achieved the targeted 75% reduction in symptoms, which is known as PASI-75, after 16 weeks. The phase 3 results just announced, however, showed only 33.1% of patients achieving PASI-75.

This represents a steep drop-off in efficacy. The worse news is that these lower levels could impact the the frequency that physicians prescribe apremilast, assuming the drug is ultimately approved. By comparison, 80% of patients taking AbbVie‘s blockbuster drug, Humira, achieved PASI-75 after 16 weeks. Clinical studies of Enbrel, which is marketed by Amgen and Pfizer , found that 46% of patients taking a 50 mg dosage of the drug achieved PASI-75 after 12 weeks.

Celgene shares traded around 2% lower on Monday morning as a result of the apremilast phase 3 results. Some investors now question whether the company will be able to hit its goal of $1.5 in annual sales for the drug.

Good news
Despite the disappointment, there is some good news. Apremilast still met its primary endpoint. Nothing has changed with Celgene’s plans regarding submitting for regulatory approval. It will submit a New Drug Application in the next few weeks for treating psoriatic arthritis and will follow up in the second half of the year for psoriasis. Celgene also intends to file for European approval for apremilast in treating both indications in the second half of 2013.

Another good sign is that the safety profile for apremilast continues to look very good. More than 96% of patients reported no adverse events or only mild-to-moderate adverse events. The most frequent of these reactions were diarrhea, nausea, and headache. 

Apremilast won’t have the efficacy of biologics already on the market like Humira and Enbrel. However, it will have some advantages. The good safety profile is one. Also, apremilast is taken in pill form instead of through injection. This convenience should sway some prescribers and patients. Then there’s the cost factor. Observers anticipate that apremilast will be considerably less expensive than its biologic rivals. 

Foolish take
I didn’t see anything with the phase 3 study that should impede the prospects for apremilast gaining approval. The lower efficacy could have some impact on sales, but the advantages of the drug — safety, convenience, and price — are still intact. My view is that apremilast should still be a blockbuster for Celgene, although sales will likely be on the lower end of the company’s forecasts.

My overall outlook on the company remains very positive. Apremilast is just one of several drugs that …read more
Source: FULL ARTICLE at DailyFinance