Zogenix Inc.’s Fintepla (fenfluramine) cleared the U.S. FDA hurdle shortly after its March 25 PDUFA date, expanding the drug’s use in patients with Lennox-Gastaut syndrome (LGS) and validating UCB SA’s $1.9 billion acquisition of Emeryville, Calif.-based firm, which closed earlier this month.
Bristol Myers Squibb Co.’s relatlimab will hit the market as the first U.S. FDA-approved LAG-3 inhibitor, cleared by the agency for use in a fixed-dose combination with Opdivo (nivolumab) to treat adult and pediatric patients with unresectable or metastatic melanoma. The approval, a day ahead of the March 19 FDA target date, adds another type of immune checkpoint inhibitor to the oncology arsenal, which already includes drugs targeting PD-1/PD-L1 and CTLA4.
Marinus Pharmaceuticals Inc.’s GABAA receptor agonist Ztalmy (ganaxolone), has won U.S. FDA approval for treating seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder.
Marinus Pharmaceuticals Inc.’s GABAA receptor agonist Ztalmy (ganaxolone), has won U.S. FDA approval for treating seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder. It is the first therapy indicated specifically for the rare genetic condition and is now approved for patients ages 2 and older, earning Marinus a rare pediatric disease priority review voucher.
Sorrento Therapeutics Inc.’s majority-owned subsidiary, Scilex Holding Co., and antibody drug developer Apexigen Inc. both announced plans late this week to go public via mergers with special purpose acquisition companies (SPACs), suggesting that, despite current market conditions, SPACs are still considered a viable option for firms seeking cash and access to public markets.
With global COVID-19 cases on the rise once again, the World Trade Organization (WTO) said it reached consensus among four WTO members – the U.S., European Union, India and South Africa – for an IP waiver regarding vaccines, a proposal to which biopharma reiterated its objections. Meanwhile, in the U.S., the White House appealed to Congress to provide adequate funding to continue the country’s pandemic response, and Pfizer Inc. and Biontech SE submitted an application to the FDA seeking emergency use authorization for a fourth shot of its COVID-19 vaccine.
Discussions with regulators on a proposed phase III trial design are up next for Bridgebio Pharma Inc., which reported positive phase II data for BBP-418 (ribitol) in patients with limb-girdle muscular dystrophy type 2 (LGMD2i), the first of several clinical readouts expected in 2022, as the Palo Alto, Calif-based company looks to regain its footing after disappointing top-line data for its phase III program in transthyretin amyloid cardiomyopathy leveled the stock late last year.
Partners Sanofi SA and Swedish Orphan Biovitrum AB (Sobi) said regulatory submissions are expected this year for once-weekly factor VIII therapy efanesoctocog alfa in hemophilia A following top-line success in a pivotal phase III study, which showed a clinically meaningful prevention of bleeds in people with severe disease receiving prophylaxis over 52 weeks. The drug, also known as BIVV-001, has fast track and orphan designations in the U.S., and the companies are banking on its extended half-life to go up against blockbuster bispecific antibody Hemlibra (emicizumab) from Roche Holding AG as well as a potential gene therapy from Biomarin Pharmaceutical Inc.
Reports of two patient deaths prompted Celyad Oncology SA to voluntarily pause a phase Ib trial testing its allogeneic CAR T-cell therapy, CYAD-101, in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) in patients with refractory metastatic colorectal cancer (mCRC), the latest safety hitch among efforts to advance the promise of CAR T beyond the first approvals in hematological malignancies.
Before taking on the role of CEO at Bluesphere Bio Inc., David Apelian would have said the most exciting work he’d done in immuno-oncology had been at Globeimmune Inc., where he’d served as chief medical officer for more than a decade, working in the field of cancer immunotherapy “back when no one believed the immune system would be something we could leverage against cancer.”
