Orladeyo Now Available to Eligible HAE Patients in Germany

Orladeyo (berotralstat), an oral therapy to prevent hereditary angioedema (HAE) attacks, is now available to patients, 12 and older, in Germany.

“We have an experienced team in place in Germany that is excited and honored to bring the first oral, once daily therapy to HAE patients in Europe,” Charlie Gayer, chief commercial officer of BioCryst Pharmaceuticals, which developed the therapy, said in a press release.

“Patients and physicians across Europe have told us about the significant need for a targeted oral therapy for HAE, and Germany is the first of many upcoming European launches for Orladeyo,” Gayer added.

The German launch comes about a month after the therapy was approved by the European Commission. Orladeyo is also currently available to eligible patients in France through a temporary authorization for use, granted by the French National Agency for Medicines and Health Products Safety.

Following the European Commission’s approval, health authorities in each European Union member state, as well as in Iceland, Norway, and Liechtenstein, decide separately whether to add the therapy to their respective public health programs, which allow patients to access treatments at low or no cost.

A similar process is ongoing in the U.K., where Orladeyo was granted marketing approval in May, but was already available to eligible patients through an early access to medicines scheme. This type of program allows patients with life-threatening or seriously debilitating conditions to have access to therapies not yet approved for commercial use.

Orladeyo is also approved in the U.S. and in Japan for similar indications.

Given as an oral capsule once daily, Orladeyo is designed to suppress the production of bradykinin, an inflammatory molecule overly produced in HAE patients, leading to sudden swelling and pain attacks.

By preventing bradykinin levels from rising too high, the therapy is expected to reduce HAE attacks, while improving patients’ quality of life and level of independence, due to its easier mode of administration compared with standard injectable medications.

Orladeyo’s regulatory approvals were supported by data from two ongoing trials — the placebo-controlled Phase 3 APeX-2 trial (NCT03485911) and the open-label Phase 2/3 APeX-S trial (NCT03472040) — which are testing the therapy in HAE patients, 12 and older.

Data from the trials showed that about a year of treatment with Orladeyo effectively and sustainably lowered the rate of monthly HAE attacks and the need for standard-of-care treatment, while improving quality of life for patients.

The therapy was considered generally safe and well-tolerated in both studies, with gastrointestinal problems being the most commonly reported adverse event. These side effects tended to occur shortly after starting treatment, typically resolved on their own, and became less frequent over time.