6 Biotechs Facing Make-or-Break FDA PDUFA Catalysts in November


October was a pretty big month for approvals by the U.S. Food and Drug Administration (FDA). Approvals included Flexion Therapeutics’ Zilretta for osteoarthritis, Neurocrine Biosciences’ supplemental approval for Ingrezza for tardive dyskinesia, and Tesaro’s Rolapitant IV for nausea and vomiting associated with chemotherapy.

There were a few notable failures as well. The FDA rejected Mylan and Biocon’s biosimilar for Amgen’s Neulasta, AcelRx Pharmaceuticals’ Dsuvia, and PTC Therapeutics’ Translarma for Duchenne muscular dystrophy (DMD). Some of those were related to manufacturing issues rather than efficacy, so we may see them approved in the future.

Here’s a look at six drugs facing FDA decisions in November.

 1. Collegium Pharmaceutical

The company has a supplementary new drug application (sNDA) coming up on Nov. 4 for Xtampza ER (oxycodone) for pain management that’s bad enough to require daily, 24/7, long-term opioid treatment where there are no other options. It was approved by the FDA in April 2016, and the company is looking to enhanced labeling. The actual date will likely be Nov. 17, because the company submitted more supportive documentation in August, which the FDA defined as a major amendment.
2. Keryx Biopharmaceuticals

Keryx also has an sNDA, in this case for Auryxia, to expand the indication to include iron deficiency anemia in patients with non-dialysis dependent chronic kidney disease. The PDUFA date is Nov. 6.

3. Bristol-Myers Squibb

Another sNDA, this for Sprycel (dasatinib). The company wants approval to treat children with Philadelphia chromosome-positive chronic phase chronic myeloid leukemia (CML) in addition to approval as a powder for oral suspension formulation. The drug was originally approved in 2006 to treat adults with Ph+ CML who are resistant or intolerant to previous therapy, including imatinib, as well as for Ph+ acute lymphoblastic leukemia (ALL). The PDUFA date is Nov. 9.

4. Dynavax Technologies Corporation

The company has submitted a biologics application (BLA) for Heplisav-B, an adult hepatitis B vaccine. The FDA has rejected the vaccine twice, on Feb. 25, 2013 and Nov. 14, 2016. The most recent rejection asked for more information related to both efficacy and safety. On Aug. 10, 2017, the FDA’s Vaccines and Related Biological Products Advisory Committee voted 12 to 1 in favor of the vaccine, at least in terms of its safety, but also commented on the post-marketing study design. The PDUFA date is Nov. 10.
5. Heron Therapeutics

Heron has a new drug application (NDA) for Cinvanthi (HTX-019) to treat nausea and vomiting caused by chemotherapy. The PDUFA date is Nov. 12. Benzinga writes, “Cinvanthi is Heron’s proprietary formulation of aprepitant, possessing the potential to be the first polysorbate 80-free, intravenous formulation of aprepitant. Aprepitant is a neurokinin-1 receptor antagonist used for preventing chemotherapy-induced nausea and vomiting.”

6. Ultragenyx Pharmaceuticals

The company’s biologics application for rhGUS (UX003) for mucopolysaccharidosis VII or Sly syndrome, has a PDUFA date of Nov. 16. In July 2016, Ultragenyx released Phase III clinical data on the drug showing it had met the primary endpoint, but the secondary endpoint was not.


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