Just-approved Orladeyo Now Being Shipped to US Patients

Just a couple of weeks after its U.S. approval, Orladeyo (berotralstat) is now available for direct shipment to hereditary angioedema (HAE) patients in the United States who hold a prescription for the oral medication.

According to BioCryst Pharmaceuticals, the maker of Orladeyo, the medicine will be provided exclusively by specialty pharmacy Optime Care, which has already begun shipping to patients.

“Access to medicine is critical to HAE patients. Many patients have been waiting for an oral option and I am very pleased that they have support from BioCryst to access Orladeyo so quickly following FDA approval,” Douglas R. Lotz, MD, senior partner of Family Allergy & Asthma, in Louisville, Ky., said in a press release.

Orladeyo is a small molecule designed to suppress the activity of plasma kallikrein, a precursor of bradykinin — an inflammatory molecule produced in excess in HAE patients, leading to sudden and recurrent bouts of swelling.

By suppressing plasma kallikrein, the medication is expected to lower bradykinin levels and prevent angioedema attacks.

Approved in early December 2020 in the U.S., Orladeyo is the first and only oral treatment to prevent swelling attacks in HAE patients, ages 12 and older. Prior to this approval, preventive treatments were available only via injections or infusions.

Similar applications are being reviewed in Japan and in the European Union, with a decision expected this month in Japan; a decision in the EU is anticipated in March 2021.

Aiming to accelerate access to treatment and smooth the journey of patients wishing to take Orladeyo, BioCryst launched a new support program, dubbed EMPOWER Patient Services. The program puts HAE patients and healthcare providers in contact with a dedicated care coordinator for financial and reimbursement assistance, and for other support tools.

Those interest can access additional information here or by calling 1-866-5-EMPOWER (1-866-536-7693).

“Our goal is to provide a best-in-class partnership that enables an individualized approach for physicians and their patients,” said Charlie Gayer, chief commercial officer of BioCryst.

“Through our dedicated care coordinators, we offer a single point of contact to assist patients and healthcare providers throughout the treatment journey,” he said. “From the transition to Orladeyo, coordination of deliveries, to ongoing patient support, EMPOWER puts the HAE patient at the center.”

A recent $325 million investment from Royalty Pharma and Athyrium Capital Management has supported the launch of Orladeyo in the U.S., and also will aid its potential launch in Europe, the company announced in another press release.

“We believe Orladeyo will be a transformative medicine and we are excited to partner with BioCryst to bring this oral, once-daily medicine to HAE patients,” said Pablo Legorreta, CEO of Royalty Pharma.

“The substantial financial commitment of exceptional long-term partners like Royalty Pharma and Athyrium Capital Management enables BioCryst to fully invest in the launch of Orladeyo,” added Jon Stonehouse, CEO of BioCryst.