EMA Rejects PTC Once More; Novartis Secures License for Cancer Treatment Developed by China-Based Biotech

Here’s a quick overview of recent developments involving PTC Therapeutics, Novartis, as well as updates from Amylyx and Intercept Pharmaceuticals that you might have missed.

The European Medicines Agency is sticking to its guns by not renewing the marketing authorization for Translarna, a medication created by PTC Therapeutics aimed at treating a rare muscle degeneration condition. Initially approved in 2014, the agency required PTC to collect further evidence for proving the drug’s effectiveness in specific instances of Duchenne muscular dystrophy. However, following four evaluations, the EMA determined that the drug’s efficacy remains unvalidated. The agency is set to relay its decision to the European Commission, which will finalize the drug’s status across EU member states. — Jacob Bell

Novartis has invested million to acquire global licensing rights for a single small molecule cancer treatment from China’s Chengdu Baiyu Pharmaceutical. The agreement could expand to .1 billion pending successful completion of developmental and sales milestones; however, details about the drug’s biological or disease targets have not been disclosed. Chengdu Baiyu has stated that it is “dedicated to researching the fundamental pharmacological elements of Ginkgo biloba,” which includes the creation of “botanical” therapeutics. The company also has six experimental cancer drugs under development. — Jonathan Gardner

Amylyx Pharmaceuticals experienced fluctuations in its stock prices Thursday following the release of data regarding one of its primary drugs. The treatment, known as AMX0035, showed positive results in a small study involving patients with Wolfram Syndrome, a rare hereditary disorder impacting organs like the eyes, brain, and pancreas. The company is planning to consult with stakeholders, including the Food and Drug Administration, regarding the next steps for Phase 3 testing, with updates anticipated next year. Although AMX0035 had received approval for ALS treatment, it was withdrawn from the market in April after a follow-up trial revealed it did not show substantial improvement compared to a placebo for the debilitating condition. — Jacob Bell

The FDA has postponed a decision regarding full approval of Intercept Pharmaceuticals’ Ocaliva for primary biliary cholangitis, a disorder for which the Alfasigma affiliate received accelerated approval in 2016. The FDA has deferred its October 15 deadline without providing a new timeline, as stated by Intercept. This delay follows a negative recommendation made by a panel of independent advisers on September 13, which concluded that the confirmatory trials did not demonstrate adequate evidence that Ocaliva effectively prevented liver transplants or death in patients with the rare disease. Ocaliva is Intercept’s sole product and generated 6 million in sales during its last complete year of operation as an independent entity in 2022. — Jonathan Gardner

The Australian pharmaceutical regulatory authority has opted not to approve Leqembi, the highly anticipated Alzheimer’s treatment by Eisai and Biogen. In a statement released Wednesday, the Therapeutic Goods Administration indicated that the safety risks associated with Leqembi surpass its potential advantages based on the current evidence. Eisai intends to request a reevaluation of this decision. While the drug has received approval in the U.S. for early-stage Alzheimer’s patients, it has faced significant resistance in Europe and now Australia, where estimates suggest over 400,000 individuals are living with dementia. — Jacob Bell

Amicus has successfully delayed competition for its main product, the Fabry disease medication Galafold, until 2037 through a settlement with Teva Pharmaceuticals. Under the terms of this agreement, Amicus will allow Teva to market a generic version of Galafold starting January 30 of that year, and both parties will withdraw any ongoing federal litigation related to Galafold’s patents. However, Amicus still faces legal challenges from another generic manufacturer, Aurobindo, with litigation currently on hold against a third competitor, Lupin. — Jonathan Gardner