2023-01-23b from pharmaceutical-technology.com

In 2022, the FDA approved only 37 new medicines, an underwhelming number compared to 98 in 2018. However, while only around 34% of the approvals in 2018 were for orphan drugs, 54% new approvals in 2022 were for drugs to treat rare diseases.

Major pharmaceutical acquisitions have taken place in recent months in the rare disease space, as the number of orphan drug approvals continues to grow. Amgen buying Horizon Therapeutics and Ipsen’s acquisition of Albireo Pharma show an increased appetite for investment in therapies for rare diseases.

This was not always the case. “Up until this century, drug companies were not particularly interested in rare diseases,” says Dr. Alan Percy, MD, a pediatric neurologist at the University of Alabama in Birmingham. “[In] the last 20 years, there have been a large number of companies that have jumped into the rare disease arena, and for some diseases like Rett syndrome, they have targeted resources to developing therapeutic agents,” he says. 

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