3 FAP Therapies Win 2020 Prix Galien USA Awards for Best Biotechnology Product

Three recently approved therapies for treating familial amyloid polyneuropathy (FAP), also known as hereditary transthyretin amyloidosis with polyneuropathy, have won their developers Best Biotechnology Product honors in the 2020 Prix Galien USA Awards, the Galien Foundation announced.

Ionis Pharmaceuticals and its subsidiary Akcea Therapeutics claimed the prize for their development of Tegsedi (inotersen), while Alnylam Pharmaceuticals was recognized for its work in developing Onpattro (patisiran). Pfizer won the award for its development of Vyndaqel (tafamidis meglumine).

The three winners in the biotechnology products category, which had a total 23 nominees, were announced at the annual Prix Galien Awards Gala, held recently at the Alexandria Center for Life Science in New York City, and via remote video links. It was the foundation’s first time holding the ceremony in a hybrid format, both in-person and digitally.

All four pharmaceutical companies were recognized for developing therapies to help manage the symptoms of FAP, and to slow down the progression of TTR amyloid deposits, which cause complications for people with the genetic disorder.

Tegsedi is the first and only one of the therapies that can be self-administered by patients at home.

“We are thrilled to accept this award for Tegsedi — a treatment that has already provided substantial benefit for those who have been devastated with the polyneuropathy associated with hereditary transthyretin amyloidosis,” Brett P. Monia, PhD, CEO and founding scientist at Ionis, said in a press release.

The medication works by blocking the production of the TTR protein, which builds up and causes damage to the peripheral nerves and hearts of FAP patients. An antisense oligonucleotide, Tegsedi binds to the messenger RNA containing instructions to make TTR and targets it for elimination, preventing these protein clumps from forming.

In addition to the  U.S. and Europe, Tegsedi is approved to treat FAP patients in several other countries. These approvals were based on data from a Phase 2/3 trial (NCT01737398) that showed that the medication was better than a placebo at lowering TTR levels, improving quality of life, and reducing neurological deficits. Importantly, the treatment can be administered by patients at home, through a subcutaneous (under-the-skin) injection.

“Our vision in the discovery and development of Tegsedi was to improve the lives of patients living with the devastating effects of this rare genetic disease that impacts the lives of generations of families,” Monia said.

“I would like to thank our teams at Akcea and Ionis for their tireless work and dedication in developing Tegsedi and giving hope to patients worldwide,” he added.

The Prix Galien USA Awards distinguish the best therapies competing in four categories: Best Pharmaceutical Agent, Best Biotechnology Product, Best Medical Technology, and Best Digital Health Product.

Candidate applications are accepted for products that have been approved by the U.S. Food and Drug Administration in the past five years and have been shown to significantly improve human health. The winners are selected by a committee composed of 17 leaders from the biomedical industry and academia; the committee includes five Nobel laureates.