Frank Vinluan

A next-generation Vertex Pharmaceuticals pain drug failed to beat a placebo in a Phase 2 clinical trial. Despite the disappointing outcome in bunionectomy pain, the company is continuing development of the oral drug in diabetic peripheral neuropathy.

Hansa Biopharma has encouraging early clinical results showing its drug imlifidase, used as a pretreatment, enabled Duchenne muscular dystrophy patients to receive the gene therapy Elevidys. Sarepta is searching for ways to mitigate excessive immune responses to Elevidys that can lead to serious and potentially fatal liver injury.

Chronic hand eczema, which can develop from chemicals and cleansers that people use frequently for work, now has its first FDA-approved treatment. The new LEO Pharma drug, Anzupgo, is a topical cream designed to block four signaling proteins that drive inflammation.

Pharmaceutical tariffs are coming, part of President Trump’s stated goal of bringing drug manufacturing back to the U.S. Untangling the sprawling, global supply chain will be complicated and expensive, and could inadvertently compete with other administration goals regarding drug pricing and availability.

Sarepta Therapeutics knew about the death of a patient treated with its experimental gene therapy for a type of limb-girdle muscular dystrophy, but said nothing publicly for a month. Financial analysts lambasted Sarepta executives for failing to disclose the fatality when the company announced a restructuring that includes stopping work on this limb-girdle program.

Sarepta’s Elevidys will remain on the market with a black box warning. But this gene therapy for Duchenne muscular dystrophy still faces commercial headwinds and analysts say the company has work to do to win over patients and investors.

The FDA published more than 200 complete response letters, the first step in what it describes as part of its effort to modernize the agency and improve transparency. But there are some caveats to the release of the FDA correspondence.

Novartis malaria drug Coartem has expanded its approval to include babies. The combination drug, originally formulated as a tablet, has a new dose strength and formulation designed for treating newborns and young infants who develop malarial infections.

KalVista Pharmaceuticals’ Ekterly is now FDA approved for treating acute swelling attacks from hereditary angioedema. The KalVista tablet provides an alternative to injectable or infused HAE medications from companies such as Takeda Pharmaceutical and CSL Behring.

Dizal Pharmaceutical’s Zegfrovy is now FDA approved for treating non-small cell lung cancer driven by a particular rare genetic signature. Johnson & Johnson drug Rybrevant already addresses this genetic signature as an earlier line of therapy, but Dizal executives contend their drug offers the potential for better safety and efficacy.