FDA Action Alert: DURECT, Vertex, Alnylam, and Regeneron


Now that July is over, the U.S. Food and Drug Administration (FDA) appears to be back in full swing with an ample docket of drug decisions to make. There are four therapeutics with PDUFA dates this week involving six biopharma companies. Here’s a look.

Cupertino, California-based DURECT Corporation and Austin, Texas-based Pain Therapeutics are waiting for an approval from the FDA by August 7 for Remoxy ER for pain. Remoxy ER (oxycodone) Extended-Release Capsules CII, based on the company’s ORADUR technology, is a long-acting formulation of oxycodone designed to discourage most methods of tampering linked to opioid abuse. On July 30, the FDA approved its New Drug Application (NDA) for Perseris (risperidone) to treat schizophrenia.

The likelihood of approval for Remoxy is slim, however. On June 26, a joint meeting of the Anesthetic and Analgesic Drug Products Advisory Committee and Drug Safety and Risk Management Advisory Committee to the FDA voted 14 to 3 against approving the drug.

Vertex Corporation has a PDUFA date of August 7 as well, for its NDA for lumacaftor/ivacaftor combination therapy for cystic fibrosis in children ages 2 to 5 years of age. The company also has a supplemental NDA (sNDA) for ivacaftor in children ages 12 to less than 24 months with a PDUFA action date of August 15. On July 30, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommended approval of the combination therapy for patients with two copies of the F508del mutation, or one copy of this CFTR gene variant plus one of 14 residual mutations.

“Our goal at Vertex is to find a cure for all people living with CF and we are moving rapidly towards treating up to 90 percent of patients,” said Reshma Kewalramani, executive vice president of global medicines development and medical affairs and chief medical officer at Vertex, in a statement. “Today’s announcement is a pivotal accomplishment along that journey.”

Alnylam Pharmaceuticals, headquartered Cambridge, Massachusetts, has a PDUFA date of Saturday, August 11 for patisiran. Patisiran is an investigational RNA interference therapy that inhibits hepatic synthesis of transthyretin. In its Phase III clinical trial in patients with hereditary transthyretin amyloidosis with polyneuropathy, the drug improved several clinical markers in the disease. Approval will also trigger a royalty entitlement “to Arbutus for the proprietary LNP technology licensed by Arbutus to Alnylam for patisiran.” The FDA had granted the drug Fast Track Designation, Breakthrough Therapy Designation, and an expanded Orphan Drug Designation for ATTR amyloidosis. The drug is also being evaluated by the European Medicines Agency (EMA).

Tarrytown, New York-based Regeneron Pharmaceuticals is awaiting a supplemental Biologics License Application (sBLA) target date for its eye drug, Eylea, of Saturday, August 11. The decision is regarding a 12-week dosing interval in wet age-related macular degeneration (AMD). Eylea is approved for wet AMD, diabetic macular edema (DME) and diabetic retinopathy (DR) in DME, and macular edema following retinal vein occlusion (RVO).

The sBLA was based on analysis of two-year data from VIEW 1 and VIEW 2 Phase III clinical trials that evaluated Eylea in wet AMD. The data found that 51 percent of patients had their Eylea dosing interval extended to every 12 weeks at the beginning of the second year of treatment and were able to maintain this every 12-week dosing interval and their best-corrected visual acuity (BCVA) improvements when they were evaluated at week 96.

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