5 Key FDA Decisions Expected in March

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Companies in the pharmaceutical industry are generally involved in the lengthy process of getting their drug candidates to market through clinical trials. There is a fair amount of risk involved, as with biotech companies, should a study come back negative or should a candidate not be approved. Conversely, if a drug is approved or passes a clinical trial, there can be big upside. U.S. Food and Drug Administration (FDA) rulings can make or break these companies. A single failed clinical trial can mean disaster for a stock.

24/7 Wall St. has collected five big FDA decisions coming up in March and added some color, along with the trading range and price target. Note that, due to many outside and internal factors, there are no assurances that the dates will not change.

As a side note about the Prescription Drug User Fee Act (PDUFA): a Priority Review designation is granted to medicines that the FDA determines have the potential to provide significant improvements in the treatment, prevention or diagnosis of a disease.

Opko Health

Opko Health Inc. (NYSE: OPK) announced that the FDA had accepted its New Drug Application (NDA) for Rayaldee for full review and set a PDUFA target date of March 29. As previously reported, this NDA is supported by data from two randomized, double-blind, placebo-controlled studies and one open-label extension study conducted in the targeted patient population at a total of 105 U.S. sites. These studies met all primary efficacy and safety endpoints.


For some background: Rayaldee has been developed for the prevention and treatment of secondary hyperparathyroidism in patients with stage 3 or 4 chronic kidney disease and vitamin D insufficiency. Rayaldee is a proprietary modified-release prohormone formulation designed to gradually correct vitamin D insufficiency.

Shares of Opko were trading at $10.21 on Friday’s close, with a consensus analyst price target of $16.80 and a 52-week trading range of $7.12 to $19.20.

Acadia Pharmaceuticals

According to Acadia Pharmaceuticals Inc. (NASDAQ: ACAD), the Psychopharmacologic Drugs Advisory Committee of the FDA will review data included in Acadia’s NDA for Nuplazid (pimavanserin) for the treatment of psychosis associated with Parkinson’s disease. The Advisory Committee Meeting is scheduled for March 29. Looking ahead, the PDUFA action date for completion of the FDA review of the Nuplazid NDA is May 1. The FDA has granted the Nuplazid NDA Priority Review status and designated it for the treatment of psychosis associated with Parkinson’s disease as a Breakthrough Therapy.

Shares of Acadia ended last week at $20.19, within a 52-week trading range of $16.64 to $51.99. The consensus price target is $45.00.

Radius Health

Now, Radius Health Inc. (NASDAQ: RDUS) is turning its focus to the completion of the work streams necessary for the submission of the NDA to the FDA for the investigational drug Abaloparatide. In the United States, the FDA requires that 12-month stability data be submitted at the time of the NDA. The 12-month time point for the abaloparatide stability samples were reached in December 2015, and Radius initiated data analysis in January 2016. To accommodate this analytic plan, the company announced a change in the NDA submission timing to the end of the first quarter of 2016 — or at the end of March.

Shares of Radius closed trading at $35.02 on Friday, with a consensus price target of $64.80 and a 52-week range of $24.75 to $84.64.

BioMarin Pharmaceutical

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) is set to report the top-line results from its Phase 3 study of pegvaliase (PEG) for the treatment of phenylketonuria by the end of the first quarter. If the data are supportive, the company plans to submit a Biologics License Application (BLA) to the FDA in the second half of 2016.

PKU is a genetic deficiency in an enzyme that prevents the breakdown of phenylalanine (Phe) in the blood. Phe is present in protein-rich foods and is an essential amino acid in the human diet, but at higher levels and if not lowered, it can lead to intellectual disability and other serious neurological problems.

On Friday, shares of BioMarin closed trading at $88.27, with a consensus price target of $119.42 and a 52-week range of $62.12 to $151.75.

Alder Biopharmaceuticals

In the first quarter, Alder Biopharmaceuticals Inc. (NASDAQ: ALDR) expects to deliver top-line data read-outs from its Phase 2b chronic migraine study and multiple dose Phase 1 study evaluating quarterly self-injectable administration of ALD403. Additionally, the company plans to initiate its next pivotal study, Promise 2, in the second half of 2016. As it continues clinical momentum for ALD403, Alder will also continue to expand its team and establish a strong foundation to support a future FDA filing and ultimately commercialization to meet the critical unmet need of the 13 million patients who are migraine prevention candidates, including the 3 million patients suffering from chronic migraine.

Shares of Alder were trading at $20.21 on Friday’s close. The consensus price target is $44.67. The 52-week trading range is $17.50 to $54.90.