Orladeyo (berotralstat) — an oral therapy developed by BioCryst Pharmaceuticals to prevent swelling attacks in people with hereditary angioedema (HAE) — is now available to patients, 12 and older, in France, following a decision by a government agency.
The French National Agency for Medicines and Health Products Safety granted a temporary authorization for use, or cohort ATU, that will allow HAE patients in the country to receive Orladeyo while the medication awaits marketing approval from the European Commission (EC). Orladeyo is also available to eligible patients in the U.K. through an early access program.
“The cohort ATU represents BioCryst’s second early access program approved in Europe and provides patients in France with faster access to treatment,” Jon Stonehouse, CEO of BioCryst, said in a press release.
This decision followed a recommendation to approve Orladeyo from the Committee for Medicinal Products for Human Use, which is part of the European Medicines Agency. Although the committee’s opinions are non-binding, they are usually followed by the EC. A final decision about the therapy’s approval in Europe is expected in the coming months.
The French cohort ATU is available to candidate medications that address high unmet medical needs, often in the case of serious or rare diseases, and whose safety and efficacy are strongly presumed. These treatments are intended for specific groups or sub-groups of patients, who are treated and monitored in accordance with defined protocols for both medicinal use and data collection.
“HAE is a debilitating and potentially deadly disease so the early access to a new treatment option for patients is exciting news,” said Laurence Bouillet, MD, PhD, head of internal medicine at the University Hospital Grenoble, Coordinating Reference Centre for HAE in France.
Orladeyo is an oral capsule, to be given once per day, containing a small molecule that prevents HAE attacks by suppressing the activity of plasma kallikrein. This protein is a precursor of bradykinin, a small protein (peptide) that is produced in excess by HAE patients, causing a series of reactions leading to inflammation, swelling, and pain.
By blocking its precursor, Orladeyo is designed to prevent bradykinin levels from rising too high and triggering attacks in these patients.
These approvals were supported by data from several clinical trials, including the Phase 3 APeX-2 study (NCT03485911). Data from this trial showed that Orladeyo significantly lowered the frequency of HAE attacks among study participants, with more than half of patients given the medication seeing their attack frequency drop by 50% or more, regardless of treatment dose.
These patients also required significantly less standard-of-care treatment and experienced more symptom-free days than those given a placebo.
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