Five Companies Face Big FDA Decisions This Month

March 14, 2016
By Mark Terry, BioSpace.com Breaking News Staff

A decision by the U.S. Food and Drug Administration (FDA) has an enormous impact, both positive or negative, on drug companies. At least five drug companies are facing big FDA decisions this month.

Opko Health

Miami, Fla.-based OPKO Health (OPK) is facing an FDA decision on the company’s oral vitamin D prohormone, Rayaldee. It is being developed to treat individuals with stage 3 or 4 chronic kidney disease (CKD). The National Kidney Foundation estimates 26 million people in the U.S. have CKD and 20 million are in stage 3 or 4. The market is estimated at $12 billion in annual sales.

The FDA has set a PDUFA target date of March 29. It has also been noted that Philip Frost, the company’s chief executive officer, recently acquired 47 million additional shares in the company since December.

Acadia Pharmaceuticals

San Diego, Calif.-based Acadia Pharmaceuticals (ACAD)’s new drug application (NDA) for Nuplazid (pimavanserin) for the treatment of psychosis associated with Parkinson’s disease, will face a decision on March 29 by the Psychopharmacologic Drugs Advisory Committee of the FDA. The Motley Fool’s Brian Feroldi has noted that this is a promising bet, but it’s also something of an all-or-nothing decision for the company, which is pretty much completely dependent on Nuplazid.

“Acadia Pharmaceuticals’ stock isn’t for the faint of heart—nearly all of its market value is riding on the success of Nuplazid,” Feroldi wrote on Feb. 22. “If the FDA gives the drug the greenlight, it’s likely Acadia’s stock will soar, but if the FDA decides to reject the drug, the company’s stock will be crushed.”

Radius Health

Waltham, Mass.-based Radius Health, Inc. (RDUS) announced a change in the NDA submission timing for its Abaloparatide to accommodate 12-month stability data.

In November 2015, Radius indicated it had sent a Marketing Authorization Application (MAA) to European regulatory agencies for Abaloparatide for postmenopausal osteoporosis. At the same time, it delayed its NDA to the FDA until the beginning of 2016.

“We felt that to rush it at the end with the NDA with a self-imposed goal might unintentionally create risk,” said Robert Ward, Radius’ chief executive officer, to BloombergBusiness. “Part of our assessment was—is this the time to ask everyone in our supply chain to work on an accelerated basis over a period of time when they all had personal plans? Or was it better for us to pick a timeline that was more respectful for what the overall work-life balance might be across our whole supply chain?”

BioMarin Pharmaceutical

San Rafael, Calif.-based BioMarin Pharmaceutical Inc. (BMRN) is reporting the topline results from a Phase III trial of pegvaliase (PEG) for the treatment of phenylketonuria by the end of March. If the data are good, it plans to submit a Biologics License Application (BLA) to the FDA in the second half of the year.

The company needs some good news. In January the FDA turned down its application for Kyndrisa (drisapersen) for the treatment of Duchenne muscular dystrophy (DMD).

Alder Biopharmaceuticals

Alder BioPharmaceuticals, located in Bothell, Wash., plans to announce topline data readouts from its Phase IIb trial of ALD403 for chronic migraine, as well as a multiple dose Phase I study evaluating quarterly self-injections of the drug. It also plans to launch the Promise 2 pivotal study in the second half of the year.

The Promise 2 trial also focuses on ALD403 in migraine. “Our focus this year remains on the accelerated development of ALD403, Alder’s pivotal-stage migraine prevention candidate with the potential to deliver migraine-free relief to patients living with migraine,” said Randall Schatzman, president and chief executive officer of Alder, in a statement. “Expected progress this year includes ongoing enrollment in our first pivotal trial, PROMISE 1 as well as top-line data read-outs later this quarter from our Phase IIb chronic migraine study and our multiple dose Phase I study evaluating quarterly self-injectable administration of ALD403…. As we continue our clinical momentum of ALD403, we will also continue to expand our 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 three million patients suffering from chronic migraine.”