Orphan product designation requests climb in the US, remain steady in the EU

Regulatory NewsRegulatory News | 11 March 2021 |  By 

The number of new requests for orphan drug designation in the United States increased 41% from 2019 to 2020, bringing the number of requests to 753 for 2020, according to the U.S. Food and Drug Administration.
There was also a significant jump in rare pediatric disease designation requests, which rose 330% from 2019 to 2020, for a total of 284 requests in 2020. The agency received 20 humanitarian use device designation requests, similar to the number received in 2019.
“Orphan drug designation is an important incentive in rare disease product development and this growth will continue to support development of treatments for rare diseases. Given the ongoing increase in volume and complexity of designations, we continue to enhance and modernize our processes,” Acting FDA Commissioner Janet Woodcock, MD, and Director of the Office of Orphan Products Development Janet Maynard, MD, wrote in a recent FDA Voices article.
They reported that the agency completed review of 100% of all new orphan drug designation requests within 90 days in 2020. Moving forward, the agency target is to complete at least 90% of all new orphan drug designation requests within 90 days.
Overall, the agency approved 32 novel drugs and biologics with an orphan drug designation in 2020. Some of the noteworthy approvals included a new treatment for Hutchinson-Gilford Progeria Syndrome and progeroid laminopathies, rare genetic conditions that lead to premature aging; a new drug for the treatment of patients with hereditary angioedema; and a chimeric antigen receptor (CAR) T-cell therapy for adults with relapsed or refractory mantle cell lymphoma.
The FDA also approved one medical device in 2020 under the Humanitarian Device Exemption program for medical devices with orphan indications.
In the European Union (EU), there were 235 applications for orphan medicines designation and 149 decisions on designation issued in 2020, compared with 233 requests and 113 decisions in 2019, according to the European Medicines Agency (EMA) annual report on the use of the special contribution for orphan medicinal products.
The clinical areas that most often received positive opinions from the European Commission’s Committee for Orphan Medicinal Products in 2020 were congenital, familial, and genetic disorders; blood and lymphatic system disorders; nervous system disorders; and neoplasms (benign, malignant, and unspecified).
Overall, the EC granted 21 new marketing authorizations for orphan medicinal products in 2020. Additionally, the EC processed fee reductions for designated products of more than 11 million Euros in 2020. The bulk of the fee reductions were for protocol assistance and follow up, followed by full applications.


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Tags: diseases, EMA, EU, FDA, rare, US

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