“We are ready to launch ORLADEYO to bring HAE patients the oral, once-daily medicine they have been waiting for to prevent attacks, reduce their burden of therapy and live a normal life,” says Jon Stonehouse, President and CEO of BioCryst Pharmaceuticals, Inc. at the announcement of financial results for the third quarter ended September 30, 2020.
Updates and Key Milestones for 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 a decision on approval 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 the build-out of the commercial infrastructure to support the successful launch of ORLADEYO in the U.S.
- The company has hired and trained accomplished U.S. rare disease sales and market access teams and has deployed a robust patient services support hub.
- The company is well-positioned in terms of product supply and inventory on-hand to support the launch and anticipated demand for ORLADEYO.
- On 30 October 2020, the company announced that the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted oral, once-daily berotralstat a positive scientific opinion through the Early Access to Medicines Scheme (EAMS). Under the EAMS, HAE patients in the UK aged 12 years and older can gain access to berotralstat for the routine prevention of recurrent attacks of HAE before the drug is granted marketing authorization by the European Commission. Medicines included in the EAMS are those that have a high unmet need, are intended to treat, diagnose or prevent seriously debilitating or life-threatening conditions where there are no adequate treatment options, and are likely to offer significant advantage over methods currently used in the UK.
- On 28 October 2020, the company announced it will present five abstracts and one Distinguished Industry Oral Abstract, including 48-week results from the APeX-2 trial and new data on quality of life and the treatment burden of injectable medication administration, at the upcoming (virtual) Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology on 13-15 November 2020.
- On 22 October 2020, the company announced that data from the first 24 weeks of the APeX-2 trial of oral, once-daily berotralstat in patients with HAE have been published online by the Journal of Allergy and Clinical Immunology.