RARE Daily

A Year of Many Firsts

January 21, 2021

The following is excerpted from NEXT 2021: A Time for Resilience and Ingenuity, Global Genes annual report on world of rare disease. The report will be released in February, but you can request a free, electronic edition here.

Last year was a productive one for rare disease drug approvals marked by many firsts.

In 2020, the U.S. Food and Drug Administration approved a total of 31 novel orphan drugs, 58 percent of the new molecular entities the agency approved during the year. That compared to 21 novel orphan drugs the agency approved in 2019, 44 percent of all new molecular entities that year.

This group of drugs represented significant innovation as 12 (39 percent) represented first-in-class therapies. Just more than half of the approvals were therapies to treat rare forms of cancer.

The approvals included Genentech’s Evrysdi, the first oral, at-home treatment for the progressive and deadly neuromuscular disease spinal muscular atrophy; Eiger Biopharmaceutical’s Zokinvy, the first drug approved for the ultra-rare disease progeria, which causes premature aging; and Horizon Pharmaceuticals’ Tepezza, a monoclonal antibody that is the first approved therapeutic for the treatment of thyroid eye disease.  

Below is a chart of the novel orphan drug therapies the FDA’s Center for Drug Evaluation and Research approved in 2020 and their various designations.

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