Orphan Drugs 2024: A Review of Progress in the US and Europe

Drug therapy plays a key role in the treatment of rare (orphan) diseases, which often have no effective treatment. On the eve of the new year, we decided to take stock and find out which orphan drugs were registered in the US and Europe in 2024.

What are orphan drugs?

• FDA (USA): Orphan drugs are those intended to treat diseases that affect fewer than 200,000 people in the US, or to treat diseases that do not generate sufficient profit to cover the costs of development and production.
• EMA (Europe): Orphan drugs are those used to treat rare diseases that are life-threatening or seriously impair quality of life. These diseases affect no more than 5 out of 10,000 people in the European Union.

Special designations and programs to accelerate the development of orphan drugs:

1. Breakthrough Therapy (FDA): Awarded to drugs for the treatment of serious or life-threatening diseases when preliminary clinical data show a significant improvement over existing treatments. Provides intensive interaction with the FDA to expedite the development process.
2. Priority Review (FDA): Reduces the standard review time for a drug application that represents a significant advance in the treatment, prevention, or diagnosis of a serious disease from 10 months to 6 months.
3. Fast Track (FDA): An FDA program to expedite the development and review of drugs intended to treat serious conditions and address unmet medical needs. Involves more frequent interactions with the FDA and the ability to submit approval documents in batches.
4. Rare Pediatric Disease (FDA): A designation given to drugs developed to treat rare diseases in children. Provides the opportunity to receive a certificate that will expedite the review of other company applications.
5. Conditional Marketing Authorization (EMA): Allows the approval of drugs based on less complete data if they are intended to treat diseases with high medical need. However, companies are required to provide additional data after approval.

This information highlights the importance of continuous work to develop new drugs to treat rare diseases and offers hope for new opportunities in the future.