Voyager Therapeutics Inc. is getting $30 million up front in a potential $630 million gene therapy deal with Pfizer Inc., the company’s first such agreement since a strategic refocusing effort earlier this year and a much-needed endorsement of a next-generation AAV capsid platform that has shown promising though early stage data.
Verrica Pharmaceuticals Inc.’s launch plans for drug-device candidate, VP-102 (cantharidin 0.7% topical solution), in the viral skin infection molluscum contagiosum were hit with another delay, as the FDA issued a complete response letter (CRL) in response to the NDA, just two days ahead of its Sept. 23 PDUFA date.
An unexpected complete response letter (CRL) from the FDA has thrown a wrench into Sesen Bio Inc.’s commercial launch plans for Vicineum (oportuzumab monatox) in high-risk BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). The company, which has spent the past few months completing its commercial build phase and hiring sales reps ahead of the Aug. 18 PDUFA date, instead must now address outstanding clinical and statistical data and analyses, in addition to CMC issues, cited in the CRL.
A three-month delay proved to be of no concern for Nexviazyme (avalglucosidase alfa-ngpt, neoGAA), Sanofi SA’s long-term enzyme replacement therapy (ERT), which gained FDA approval for intravenous infusion to treat patients 1 and older with late-onset Pompe disease.
Manufacturing deficiencies were cited in the FDA’s complete response letter (CRL) for Spectrum Pharmaceuticals Inc.’s Rolontis (eflapegrastim), a decision that will further delay to market in the U.S. what could be the *first novel G-CSF drug in more than 15 years to treat chemotherapy-induced neutropenia.
Albireo Pharma Inc.’s Bylvay (odevixibat) becomes the first therapy to hit the commercial market for rare genetic liver disease progressive familial intrahepatic cholestasis (PFIC). A day after the European Commission approved the drug, the FDA gave the nod to the ileal bile acid transport inhibitor on its July 20 PDUFA date following a priority review.
More than four months after its original PDUFA date of Feb. 2, 2021, Mallinckrodt plc’s Stratagraft gained FDA approval for use in deep partial-thickness thermal burns.
It took a few years and three tries, but Heron Therapeutics Inc. finally got its pain drug, HTX-011, now branded Zynrelef, over the FDA finish line. Even so, the approval comes with a less broad label than Heron would have liked. Zynrelef (bupivacaine and meloxicam), which had a May 12 PDUFA date, is approved for use in adults for soft tissue or periarticular instillation to produce postsurgical analgesia for up to 72 hours after bunionectomy, open inguinal herniorrhaphy and total knee arthroplasty.
In an unusual move, three members of the Nov. 6 FDA advisory committee that voted against recommending approval of Biogen Inc.’s high profile Alzheimer’s disease candidate, aducanumab, have doubled down on their argument in an editorial published in JAMA on March 31.
