OrladeyoHereditary angioedema (HAE) type I and II – for prevention of acute attacks
New molecular entity
Development and Regulatory status
Aug 21Has been available in the US since Dec 2020 .
May 21Approved in UK for the routine prevention of recurrent hereditary angioedema (HAE) attacks in HAE patients 12 years and older. Decisions from NICE and SMC are anticipated Q4 21 .
May 21Approved in EU for prevention of acute attacks in hereditary angioedema in patients 12 years and older. A MAA has been submitted to the MHRA .
Mar 21MAA submitted under the MHRA’s new European Commission Decision Reliance Procedure .
Feb 21Recommended for EU approval by CHMP - the full indication is "for routine prevention of recurrent attacks of hereditary angioedema (HAE) in adult and adolescent patients aged 12 years and older." It should be prescribed by physicians experienced in the treatment of hereditary angioedema. .
Dec 20The FDA has approved oral, once-daily berotralstat for prophylaxis of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older .
Nov 20MHRA issues EAMS scientific opinion to BioCryst UK Limited for berotralstat, indicated for routine prevention of recurrent attacks of HAE in adults and adolescents aged 12 years and older .
Mar 20Filed in EU via centralised procedure .
Feb 20The FDA has accepted and filed its new drug application (NDA) for the approval of oral, once daily berotralstat (BCX7353) for the prevention of hereditary angioedema (HAE) attacks .
May 19BioCryst announce plans to submit a NDA to the US FDA in Q4 2019 and a Marketing Authorization Application to the EMA in Q1 2020.
Jul 18Has orphan drug status in US .
May 18MHRA grants Promising Innovative Medicine (PIM) status. Also positive opinion by EMA for orphan drug status .
Small-molecule human plasma kallikrein inhibitor which inhibits isolated human plasma kallikrein activity
HAE affects 1 in 50,000 to 100,000 people of any ethnic group and of either gender. According to the UK population mid-year estimate 2016, there are 65,648,100 people in the UK .
Hereditary angioedema (HAE) type I and II – for prevention of acute attacks
Trial or other data
Nov 20Orally administered BCX7353 will compete with Takhzyro (lanadelumab-flyo) which is a kallikrein inhibitor that is given by s.c. injection. In PIII data that led to its approval, pts treated with Takhzyro experienced a “statistically significant reduction in mean HAE attack frequency of 87% vs. placebo .
Oct 20In the APeX-2 trial (n=121), berotralstat was associated with a reduction in the rate of investigator-confirmed hereditary angioedema attacks versus placebo (2.35 attacks/month) at both 110mg (1.65 attacks/month; p=0.024) and 150mg (1.31 attacks/month; p<0.001) doses .
May 19Data from the double-blind, placebo-controlled PIII APeX-2 trial (n=121) for prevention of HAE attacks showed that BCX7353 (both 110mg and 150mg) achieved its primary endpoint (rate of HAE attacks over 24 weeks) vs. placebo. The 150mg dose reduced the HAE attack rate by 44% vs. placebo (p5% of pts in APeX-2 were nausea, dyspepsia and diarrhea.
Sep 18Results from the ZENITH-1 trial (NCT03240133) showed that a single 750 mg oral dose of BCX7353 was well tolerated and superior to placebo (p<0.05) against the majority of efficacy endpoints evaluated in HAE patients suffering an acute attack. compared to placebo, improvement in symptoms and VAS scores was seen as early as one hour after oral BCX7353 dosing, and was sustained through 24 hours..
Feb 18PIII APeX-2 trial to evaluate the efficacy of two capsule doses of BCX 7353 (110mg and 150mg) administered orally to reduce the frequency of attacks in patients with type I and type II HAE starts (NCT03485911). The primary endpoint of the trial is to determine the rate of angioedema attacks over 24 weeks of study drug administration. 96 participants will be recruited in the US, Canada, United Kingdom, Macedonia and Romania. Collection of primary outcome data due to complete May 19 .