FDA's White Oak Building

Can the FDA keep the momentum going for rare disease drug approvals?

Following a year filled with several approvals for rare disease drugs, experts are expecting this trend to continue in 2023.

By Akosua Mireku, Pharmaceutical Technology

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. Read more …