This week in drug approval news:
The FDA is making a push to speed up the review process for drugs that treat rare diseases and cancers. But pharma companies that pursue these new pathways will have to think about pricing and the potential for scrutiny as they move forward.
Makary’s plan would allow the agency to approve drugs based on surrogate endpoints, but he also wants to require that they complete confirmatory trials to prove they actually deliver clinical benefit to patients. He argued that this will help reduce the costs of drug development and lower prices for medicines in the U.S., where prescription medication costs are two-to-three times higher than in other developed countries.
Alyftrek, from J&J, is a subcutaneous formulation of the drug Trikafta that treats metastatic gastrointestinal stromal tumors (GIST), which are a type of cancerous growth in the stomach or colon. Alyftrek improves on Trikafta in several ways, including a simplified dosing regimen and the addition of a monthly injection.
Another drug to watch is Ionis Pharmaceuticals’ Donidalorsen, which targets hereditary angioedema (HAE). The disease results in recurrent episodes of severe swelling throughout the body. The drug is the first-in-class to address this condition, which can be dangerous if it affects the airways. It is the latest in a series of breakthrough treatments to target rare diseases, illustrating how the Trump administration’s policy changes are shifting the dynamic around accelerated approvals. Staying ahead of industry trends is the key to success in a complex pharmacy landscape. That’s where Serve You Rx comes in.