Key takeaways:

• Berotralstat now has two FDA-approved oral formulations for patients aged 12 years and older: capsule and pellet.
• The pellet formulation is also approved for those aged 2 to less than 12 years.

The FDA approved berotralstat oral pellets for prophylactic use in patients aged 12 years and older with hereditary angioedema, according to a press release from BioCryst Pharmaceuticals.

As Healio previously reported, the once-daily 150 mg oral pellet formulation of berotralstat (Orladeyo; BioCryst Pharmaceuticals) has already been approved for prophylactic use in patients aged 2 to less than 12 years with hereditary angioedema (HAE).

Berotralstat pellets and capsules are FDA-approved oral formulations for patients aged 12 years and older with HAE.

The other available formulation of berotralstat is a capsule, which the FDA approved back in December 2020 as a prophylaxis for HAE attacks in patients aged 12 years and older.

“The availability of 150 mg Orladeyo pellets expands BioCryst’s portfolio of targeted oral prophylactic therapyoptions to meet patients’ individual needs and preferences and reflects our commitment to optimizing the management of HAE with proven therapies that help address the evolving needs of patients across the treatment landscape,” Charlie Gayer, MBA, president and CEO of BioCryst, told Healio.

The release highlighted that the pellet formulation caters to patients who may have difficulty swallowing.

Sprinkle-like in appearance and size, the oral pellets can be poured directly into the mouth and swallowed immediately with water or milk, according to the release. They can also be sprinkled over a spoonful of soft, non-acidic food.

“Orladeyo oral pellets work the same way that is proven to be effective in the capsule formulation for ages 12 and older, in smaller pellets that can easily be swallowed,” Gayer said.

For the pellet formulation approval in adolescents and adults, BioCryst noted that the FDA’s decision was based on the comparison of the pellet to the capsule in terms of chemistry, manufacturing and controls information, as well as data demonstrating safety and tolerability.

New data on berotralstat from an updated interim analysis of the open-label APeX-P study were presented in a poster at the 2026 European Academy of Allergy and Clinical Immunology Annual Congress in June. This study included 29 patients aged 2 to less than 12 years weighing at least 12 kg with HAE due to C1-inhibitor deficiency who received either the capsule (n = 7) or pellet (n = 22) formulation.

When compared with the median adjusted rate of HAE attacks requiring on-demand treatment in the standard of care period, researchers observed a reduction in the 48-week berotralstat period (0.169 attacks/month vs. 0.691 attacks/month).

Switching to HAE attacks requiring professional care (physician office, hospital ED, urgent care and professional home care), 22 occurred in the standard of care period. With berotralstat, this number dropped to three between day 1 and week 12 of treatment, three between week 13 and week 24, three between week 25 and week 36, and zero between week 37 and week 48.

For more information:

Charlie Gayer, MBA, can be reached at allergy@healio.com.