Pharmaceutical Industry Roundup: BioCryst Divests European Business, FDA Vaccine Policy Criticized, and Clinical Trial Updates

BioCryst Sells European Orladeyo Business for $250 Million
BioCryst Pharmaceuticals has announced the sale of its European business for its hereditary angioedema drug Orladeyo to Italy's Neopharmed Gentili. The deal, valued at $250 million upfront with potential additional milestone payments of $14 million, includes the transfer of BioCryst's European sales organization to Neopharmed.
The strategic move is set to significantly improve BioCryst's financial position. The company plans to use the proceeds to pay off approximately $249 million in term debt owed to Pharmakon Advisors, resulting in an estimated $70 million savings in interest. Additionally, the sale is expected to reduce annual operating costs by $50 million.
As a result of this transaction, BioCryst has revised its financial outlook, now projecting to end 2027 with $700 million in cash, a substantial $400 million increase from previous guidance.
Former FDA Official Criticizes New COVID-19 Vaccine Policy
Peter Marks, the former head of the FDA's vaccine review division, has publicly criticized the agency's new rules for COVID-19 vaccines. In a commentary published in the New England Journal of Medicine, Marks argued that the FDA is overstepping its authority and encroaching on the domain traditionally held by the Centers for Disease Control and Prevention (CDC).
The new FDA policy requires that any new COVID-19 booster for healthy, non-elderly adults and children must be supported by large, randomized controlled trials evaluating death or hospitalization before approval. Marks contends that this approach ignores existing safety data for COVID-19 vaccines and the ongoing hospitalizations and deaths caused by the virus.
Marks, who left the FDA following disagreements with health secretary Robert F. Kennedy Jr., expressed concern that these changes were implemented without public discussion or consultation with outside public health experts who typically advise the FDA and CDC.
Clinical Trial Updates: Mixed Results for Liver Disease and Genetic Disorder Treatments
Altimmune reported mixed results from a Phase 2 trial of its experimental liver disease drug, pemvidutide, for the treatment of metabolic dysfunction-associated steatohepatitis (MASH). The drug met one of its two primary endpoints, with 59% of participants receiving a 1.2 mg weekly dose and 52% receiving a 1.8 mg dose achieving MASH resolution and no increase in fibrosis score, compared to 19% in the placebo group. However, pemvidutide failed to meet the second goal of fibrosis improvement without worsening of MASH.
In other clinical news, Belgian pharmaceutical company UCB announced positive results from a late-stage study of fenfluramine as an add-on therapy for patients with a rare genetic disorder affecting brain development. While detailed data were not released, UCB stated that the drug met the study's main effectiveness goal and was "generally well tolerated." The company plans to submit fenfluramine for regulatory approval in this new indication "as soon as possible."
Fenfluramine, notably one of the components of the controversial "fen-phen" diet regimen, is already approved in the United States as an oral solution for treating Dravet syndrome and Lennox-Gastaut syndrome, two severe seizure disorders.
References
- Biocryst sells Europe business; Peter Marks criticizes new FDA vaccine rules
Biocryst is offloading part of its business for $250 million. Meanwhile, the former FDA leader took issue with a new policy for COVID-19 shots.
Explore Further
What are the key terms of the sale between BioCryst Pharmaceuticals and Neopharmed Gentili?
How does the divestment of BioCryst's European business impact its competitive position in the hereditary angioedema drug market?
What is the background and profile of Neopharmed Gentili in the European pharmaceutical industry?
Are there major competitors of BioCryst that are engaging in similar business divestment strategies?
What implications might the FDA's new COVID-19 vaccine policy have on biotech companies' development strategies?