BioCryst Gets FDA Approval To Treat Rare Childhood Swelling Disorder With Oral Drug

The U.S. Food and Drug Administration (FDA) on Friday approved BioCryst Pharmaceuticals Inc.’s (NASDAQ:BCRX) New Drug Application (NDA) for the use of an oral pellet formulation of once-daily Orladeyo (berotralstat) for prophylactic therapy in pediatric patients with hereditary angioedema (HAE) aged 2 to <12 years.

HAE is a rare genetic disorder marked by recurring episodes of severe swelling. Symptoms often begin early, with about 40% of patients experiencing their first attack by age 5.

Orladeyo is now the first and only targeted oral prophylactic therapy for patients with HAE aged two and above.

The new Orladeyo 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.

A capsule formulation of Orladeyo received FDA approval for prophylaxis to prevent HAE attacks in adult and pediatric patients 12 years and older in December 2020.

Also Read: BioCryst Says Astria Therapeutics $700 Million Deal Gives ‘Perfect Second Product Candidate’

The approval was supported by positive interim data from the APeX-P clinical trial, the largest trial to date evaluating a long-term prophylactic therapy for HAE in patients 2 to <12 years of age.

The primary objective was to describe the PK parameters of berotralstat, and secondary objectives were to assess the safety and tolerability of berotralstat and to summarize the efficacy of berotralstat in pediatric patients with HAE.

Interim results from APeX-P showed Orladeyo was well tolerated, demonstrated a consistent safety profile across this age group, and resulted in early and sustained reductions in monthly attack rates with no new safety signals identified beyond those previously described in prior adult and adolescent trials.

BioCryst has filed its application for the use of Orladeyo oral pellets in patients with HAE aged 2 to <12 years with the European Medicines Agency and the Japan Pharmaceutical and Medical Devices Agency. Additional regulatory filings are planned in other global territories, including Canada.

BCRX Price Action: BioCryst Pharma shares were down 2.57% at $7.38 at the time of publication on Friday, according to Benzinga Pro data.

Read Next:

• Trump’s American Bitcoin Valuation Turns Attractive After Roth Capital Initiates ‘Buy’: Value Score Spikes

Photo: jittawit21/Shutterstock