BioCryst Pharmaceuticals Inc. (NASDAQ:BCRX) established expanded access program (EAP) for its investigational drug berotralsat in the treatment of hereditary angioedema (HAE)patients in the US.
Berotralstat give expanded access for treatment of HAE
With the EAP, physicians will now request the once per day oral berotralstat for patients with HAE who cannot access the drug through clinical studies. The US FDA grants expanded access as a potential pathway for patients with immediate life-threatening diseases or conditions. This helps them in gaining access to investigational drugs for treatment for those not in clinical trials. It is granted when there is no satisfactory or comparable alternative therapy option available.
Following the expanded access for the drug requests for access will have to be from a licensed US physician. Requests can be made through email via [email protected]. A new drug application for the investigational drug, berotralstat, is still under review by the US FDA. An action date under the PDUFA has been set on December 3, 2020.
BioCryst announce findings on the survey in HAE patient treatment experiences
Recently the company reported the findings of two patient surveys carried to understand the current HAE treatment experiences, expectations, and satisfaction of patients. According to patient-reported attack history, those patients that received injectable or infused prophylactic treatments (Haegarfda, Takhzyro, Cinryze) had breakout attacks on average ranging between 0.9 and 1.8 attacks in the three months before the survey. Interestingly most of the surveyed patients indicated that they expected to experience no attacks at all even after receiving prophylaxis therapy.
The company presented the data at the European Academy of Allergy and Clinical Immunology (EAACI) virtual congress. Jinky Rosselli, the company’s VP global business analysis and operation, conducted the research. Rosselli established that although the infused/injectable medication given in the last 12 years offered prophylactic attack regulation in HAE Patients, most of them still experience attacks. Further, Rosselli established that HAE patients showed interest in new preventive treatment alternatives, most of which stated they would wish to have an oral alternative.