Immedica granted orphan designation for glycerol phenylbutyrate in STXBP1-related disorders
Immedica, a pharmaceutical company headquartered in Sweden, recently announced that the European Commission granted an orphan designation for glycerol phenylbutyrate in STXBP1-related disorders. This is the first orphan designation for a medication for STXBP1 in the world!
What is an orphan designation? Does it mean the drug is approved?
In the EU, an orphan designation can be granted for a medication by the European Commission after review by the European Medicines Agency. This means that the medication has potential, but orphan designation is not a drug approval or marketing authorisation. After an orphan designation, clinical trial studies are needed to advance to a drug approval.
An orphan designation can be granted to medicines that treat, prevent, or diagnose a life-threatening or chronically debilitating rare disease, with a prevalence in the EU of below 5 in 10,000, and with either no satisfactory method of diagnosis, prevention of treatment or with significant benefit to those affected by the disease.
More information on orphan designations is here. In the US, there is a similar but independent program called an orphan drug designation. The US designation would be granted by the US Food and Drug Administration. Both of these programs provide incentives for biopharma and other drug developers to encourage development of these medicines for rare diseases.
We are hopeful for more news soon on how Immedica intends to advance development of glycerol phenylbutyrate in STXBP1.
This is a great milestone for the STXBP1 community. This also highlights the importance of the activation of our community, and the need to continue to build clinical trial readiness including participating in registries and natural history studies. Our community's work is encouraging biopharma investment!
