BioCryst advances pediatric HAE treatment as new CEO takes the helm

Durham-based BioCryst Pharmaceuticals Inc. is advancing care for children living with hereditary angioedema (HAE) as it begins a new chapter under a new CEO. The company recently received approval from the U.S. Food and Drug Administration for an oral pellet formulation of ORLADEYO to help prevent HAE attacks in children ages 2 to under 12.

HAE is a rare genetic disorder that causes sudden and severe swelling that can be painful and sometimes life-threatening. It often shows up in childhood, with around 40% of children with HAE having their first attack by age 5.

Before this approval, the only targeted treatments for children under 12 with HAE were delivered intravenously or through subcutaneous injection. As a result, roughly half as many children under age 12 with HAE receive prophylactic therapy compared with adults.

“The approval of ORLADEYO as the first and only targeted oral prophylactic therapy for patients with HAE aged 2 and above extends the benefits of oral prophylactic therapy to a vulnerable and important part of the HAE community,” said Charlie Gayer, who became CEO of BioCryst Pharmaceuticals on January 1.

The new ORLADEYO oral pellet formulation is sprinkle-like in appearance and size and can be poured directly into the mouth and swallowed immediately with water or milk, or sprinkled over a spoonful of soft, non-acidic food.

In December 2020, the capsule formulation of ORLADEYO received FDA approval to prevent HAE attacks in patients 12 years and older and is now approved in more than 45 countries.

Positive clinical findings

Approval of the ORLADEYO oral pellets follows positive interim data from the APeX-P clinical trial, which showed that the treatment was well-tolerated and had a consistent safety profile across this age group.

The oral pellet formulation led to early and lasting reductions in the number of monthly attacks in children aged 2 to under 12 and had no new safety concerns beyond those already reported in adult and adolescent studies.

BioCryst has filed regulatory applications for ORLADEYO oral pellets for the treatment of HAE in children ages 2 to under 12 with the European Medicines Agency and Japan’s PMDA, and plans additional filings in other global markets, including Canada.

Gayer, who succeeds Jon Stonehouse as CEO, spearheaded the successful launch and international rollout of ORLADEYO for those 12 years and older as BioCryst's chief commercial officer. He joined BioCryst in 2015 as vice president of global strategic marketing after spending time at Talecris Biotherapeutics and GlaxoSmithKline.

“I’m proud to have spent the past twenty-five years of my career in North Carolina,” said Gayer. “During this time, Biocryst has become a successful commercial company, thriving in the Triangle’s biotech community.”

More HAE treatment options

Gayer also played an instrumental role in the company’s recent acquisition of Astria Therapeutics, Inc. This added navenibart, a potential long-acting injectable with a 3- or 6-month administration schedule for the prevention of HAE, to BioCryst’s HAE treatment portfolio.

“The addition of navenibart fits seamlessly with our HAE core competence and could enable us to expand effective HAE treatment options, regardless of administration preference,” said Gayer.  “By addressing both parts of the market with a leading oral and potentially best-in-class injectable therapy for HAE, we are positioned to drive sustainable growth and profitability while optimally serving the HAE patient community.”

Navenibart is currently in phase 3 testing and on pace to fully enroll this year with topline data early next year. If successful, BioCryst will file with the FDA by the end of 2027 and launch the treatment by the end of 2028.

“Because of our success with ORLADEYO, we are positioned to not only advance our own discovery and commercialization but to grow through acquisitions,” added Gayer.

He says that this strategy allows the company to apply its skills in other synonymous rare disease therapeutic areas, with a focus on late-stage research for self-administered and chronic therapies that treat rare diseases ranging from patient populations with a few thousand to tens of thousands.

Expanding care for HAE and other rare diseases

The company’s HAE portfolio was spotlighted with nine presentations at the 2026 American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting held in Philadelphia February 27-March 2.

“The presentations featured new clinical trial and real-world outcomes with ORLADEYO as well as new clinical trial outcomes with navenibart,” said Gayer. “The breadth of data reflects the continued evolution of our strategy to expand and diversify our HAE portfolio in ways that matter to patients and their care teams.”

Looking ahead, Gayer says that the company’s goal is to grow its portfolio of therapies for rare diseases. It is prioritizing the development of BCX17725, a KLK5 inhibitor protein therapeutic for the potential treatment of Netherton syndrome. This rare genetic disease is characterized by uncontrolled skin shedding and has no approved therapies.

BCX17725 is currently in Phase 1b in healthy volunteers. By the end of the year, up to a dozen patients will have completed three months of therapy. If successful, BioCryst plans to initiate a pivotal study in 2027.

Read more about BioCryst Pharmaceuticals:

BioCryst Pharmaceuticals expands HAE portfolio with acquisition of Astria Therapeutics

FDA accepts BioCryst’s application to expand ORLADEYO to young HAE patients