Mixed opinions from the U.S. FDA’s Oncology Drugs Advisory Committee last month didn’t stop the agency from green-lighting an expanded label for Abecma (idecabtagene vicleucel) to include adults with relapsed or refractory multiple myeloma (r/r MM) after two or more prior lines of therapy including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 monoclonal antibody.
Less than a month after disclosing that its confirmatory phase III trial of Relyvrio (sodium phenylbutyrate plus taurursodiol) fell short of its endpoint, Amylyx Pharmaceuticals Inc. is withdrawing the amyotrophic lateral sclerosis (ALS) drug from the market.
In a long and winding regulatory road that began with two complete response letters 15 years ago, Allschwil, Switzerland-based Basilea Pharmaceutica Ltd. finally gained U.S. FDA approval of its intravenous cephalosporin antibiotic, Zevtera (ceftobiprole medocaril sodium), to treat Staphylococcus aureus bacteremia, acute bacterial skin and skin structure infections (ABSSSI) and community-acquired bacterial pneumonia.
The lingering effects of the COVID-19 pandemic and ongoing recruitment/retention issues are making it difficult for the U.S. FDA’s bioresearch monitoring program to keep up with the on-site clinical research inspections that are a cornerstone of the preapproval process for new drugs, biological products and medical devices. The resulting delays could threaten the approval timelines for many products.
Building D&D Pharmatech Inc. has been a rollercoaster ride, according to CEO Seulki Lee. The U.S. and Korea-based biotech is on another ascent, having scored U.S. FDA fast track designation for its metabolic dysfunction-associated steatohepatitis (MASH) drug, ahead of its third attempt at a public listing.
The U.S. FDA accepted for review Daiichi Sankyo Co. Ltd.’s and Astrazeneca plc’s BLA for datopotamab deruxtecan to treat adults with unresectable or metastatic hormone receptor-positive, HER2-negative breast cancer who have received prior systemic therapy for unresectable or metastatic disease.
Voydeya (danicopan), from Alexion, Astrazeneca Rare Disease, racked up its second global approval as the U.S. FDA greenlit it as an add-on therapy for treating extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (PNH), a crowded market with several already approved treatments and more in development.
Trouble continues to dog Verve Therapeutics Inc.’s base editor of the PCSK9 gene, VERVE-101, so the company paused enrollment in a phase Ib study in cholesterol lowering to focus on the similarly designed VERVE-102.
Beijing- and Shanghai-based Sperogenix Therapeutics Ltd. said that China’s regulatory agency accepted the NDA filing and granted priority review of Agamree (vamorolone) for Duchenne muscular dystrophy on March 26.
After a complete response letter (CRL) from the U.S. FDA in 2022 and approvals in Europe, Japan and Australia, Akebia Therapeutics Inc.’s Vafseo (vadadustat) has finally been approved by the FDA. But the uphill climb still isn’t over for Akebia, as it has more hurdles to clear for the drug.