“We are currently in an exciting transformation from a company primarily focused on R&D to one that is about to launch its first oral drug to patients with HAE (ORLADEYO) this year. We expect to end the year with ORLADEYO approved in the U.S. and Japan”, says President and CEO Jon Stonehouse, BioCryst Pharmaceuticals, Inc., at the presentation of the financial results for the second quarter of 2020.
HAE Program – ORLADEYO: Oral, once-daily treatment for prevention of HAE attacks
- BioCryst expects three regulatory approvals for ORLADEYO in Q4 2020 and early 2021.
The U.S. Food and Drug Administration (FDA) is reviewing a new drug application for ORLADEYO and has set an action date of 3 December 2020, under the Prescription Drug User Fee Act (PDUFA).
- In Japan, ORLADEYO is being reviewed under Sakigake designation. The Pharmaceutical and Medical Devices Agency (PMDA) has confirmed their regulatory review schedule and the company expects an approval decision in December 2020.
- On 30 March 2020, the company announced that the European Medicines Agency (EMA) had validated its marketing authorization application (MAA) submission for ORLADEYO and begun its formal review of the MAA under the centralized procedure. The company expects an opinion from the Committee for Medicinal Products for Human Use (CHMP) within approximately 12 months from MAA validation.
- BioCryst has completed significant preparations to support the launch of ORLADEYO in the U.S.
- The company has attracted an accomplished U.S. rare disease sales team, which averages 20 years in pharmaceutical sales and nearly a decade of rare disease experience.
- The company is well-positioned in terms of product supply and inventory on-hand to support the launch and anticipated global demand for ORLADEYO.
- On 9 June 2020, the company announced that it had established an expanded access program with ORLADEYO for patients with HAE in the United States. Through this program, physicians may be able to request ORLADEYO for HAE patients who do not have access to the product through a clinical trial.
- On 6 June 2020, at the European Academy of Allergy and Clinical Immunology (EAACI) Digital Congress, the company presented new data highlighting the burden of therapy faced by HAE patients taking currently available injectable prophylactic medication. Patients taking oral, once daily ORLADEYO experienced sustained decreases in their attack frequency and improvements in quality of life scores over 48 weeks. ORLADEYO was also safe and generally well-tolerated over 48 weeks in both the APeX-2 and APeX-S clinical trials.
“Our ongoing dialogue and research with HAE patients and physicians continue to reinforce their strong demand for an oral, once-daily medicine that is safe and provides the significant and sustained attack reduction we are seeing with ORLADEYO in our clinical program. Nearly half of patients in our APeX-2 and APeX-S trials have prior experience with injectable or infused therapies and most have chosen to remain on ORLADEYO,” said Charlie Gayer, chief commercial officer of BioCryst.