Updates provided on berotralstat and navenibart

[Berotralstat is often referred to by its brand name, Orladeyo]

A series of business updates relating to berotralstat, which is a currently available HAE treatment, and a potential new HAE medicine navenibart, were provided by the pharmaceutical company BioCryst. The key updates shared were:

  • In October 2025, the company completed the sale of its European Orladeyo business to Neopharmed Gentili SpA.
  • In December 2025, the company received FDA approval for the Orladeyo pellet formulation for patients ages 2 to <12 with HAE, making it the first and only targeted oral prophylactic therapy for children living with HAE.
  • In January 2026, the company completed its acquisition of Astria Therapeutics Inc. Through the transaction, BioCryst added navenibart to its HAE portfolio. Navenibart is an injectable, long-acting, monoclonal antibody inhibitor of plasma kallikrein currently in Phase 3 clinical development for HAE prophylaxis.
  • The navenibart Phase 3 program is currently enrolling and is on track to support regulatory filing by the end of 2027. The program consists of the ALPHA-ORBIT Phase 3 trial and the ORBIT-EXPANSE long-term trial. ALPHA-ORBIT is a randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of navenibart over a six-month treatment period, with dosing arms every three months (Q3M) and every six months (Q6M). After completing ALPHA-ORBIT, eligible participants may continue into the ORBIT-EXPANSE trial, in which all participants will receive navenibart in either Q3M or Q6M regimens.
  • New positive, interim results from the long‑term, open‑label ALPHA‑SOLAR trial show sustained, robust HAE attack suppression with navenibart administered every three and six months. The mean and median HAE attack rate reductions from baseline were 92% and 97% in the Q3M dosing arm and 90% and 97% in the Q6M dosing arm. Long-term data up to 24 months show durable efficacy and a favorable safety profile for both dosing regimens. BioCryst will present these results in a late-breaking presentation, and other abstracts, at the 2026 American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting in Philadelphia, 27 February – 2 March 2026.

Charlie Gayer, President and Chief Executive Officer of BioCryst, said: “2025 was fundamentally transformative for BioCryst. We also advanced key business development initiatives that streamlined our operations and further strengthened our leadership position in hereditary angioedema. We entered 2026 with strong momentum, completing the acquisition of Astria Therapeutics to grow our HAE portfolio to meet the needs of more patients, while adding to our long-term growth trajectory well into the next decade.”

(Source: BioCryst)