The EMA is standing firm on its refusal to recommend approval of the amyotrophic lateral sclerosis (ALS) treatment Albrioza in Europe after re-examining Amylyx Pharmaceuticals Inc.’s marketing authorization application and remaining unconvinced that the main study demonstrated the drug effectively slows disease progression.

“We’re going to battle,” PTC Therapeutics Inc. CEO Matthew Klein said, responding to a surprise negative opinion from the EMA’s Committee for Medicinal Products for Human Use on converting the conditional marketing authorization to full status for Translarna (ataluren) in the treatment of nonsense mutation Duchenne muscular dystrophy. The opinion applies to the renewal of the existing conditional authorization, too.

Supporting their conclusions with data from the same phase III study, the EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion for extending the use of Oncopeptides AB’s Pepaxti (melflufen) to earlier lines of treating relapsed, refractory multiple myeloma even as the FDA dug in its heels about withdrawing the drug from the U.S. market.

Avencell Therapeutics Inc. has received approval from the EMA for its clinical trial application (CTA) for AVC-201 for the treatment of relapsed/refractory acute myeloid leukemia (AML) and other selected hematologic malignancies positive for CD123. A phase I study will be conducted in Germany.

Mirati Therapeutics Inc.’s cancer treatment Krazati (adagrasib) is having a tough time outside the U.S. The EMA’s Committee for Medicinal Products for Human Use (CHMP) came out with a negative opinion on an MAA for treating KRAS G12C-mutated advanced non-small-cell lung cancer (NSCLC). The ruling came with a flurry of positive CHMP opinions for companies, including Abbvie Inc., Beigene Ltd. and the Janssen Pharmaceutical Cos., plus a mixed result for Ipsen SA after a stumble earlier this week.

The EMA and the European Center for Disease Control have said COVID-19 vaccine manufacturers should ditch the existing formulations and adapt their products to target the omicron-descendant XBB.1.5, to protect against SARS-CoV-2 in the winter of 2023–2024.

Otsuka Pharmaceutical Co. Ltd. has gone back on efforts to get its medicine, Asimtufii (aripiprazole), a long-acting maintenance treatment for schizophrenia, approved in Europe, after the EMA gave a provisional negative opinion.

Otsuka Pharmaceutical Co. Ltd. has gone back on efforts to get its medicine, Asimtufii (aripiprazole), a long-acting maintenance treatment for schizophrenia, approved in Europe, after the EMA gave a provisional negative opinion.

A heart-protective cardiac myosin inhibitor and two biologics – one for a type of non-Hodgkin lymphoma and another for an inflammatory skin condition – were among the therapies recommended for approval by the EMA’s Committee for Medicinal Products for Human Use this week.