Ascendis TransCon CNP receives Orphan Drug Designation

On the Feb 28th  2019, Ascendis announced that the innovative medicine

TransCon CNP was granted Orphan Drug Designation in achondroplasia by the FDA.  The company is dedicated to doing everything possible to advance its achondroplasia program.  For this reason, Ascendis is conducting the ACHieve Study, a natural history study, and working to initiate the phase 2 trial later 2019. You can read the full press release at Ascendis website here.

What is the meaning of having an Orphan drug designation?

The Food and Drug Administration (FDA) is an agency within the U.S. Department of Health and Human Services.

With the Orphan Drug Act (ODA), the FDA provides for granting special status to a drug or biological product (“drug”) to treat a rare disease or condition upon request of a sponsor (usually a pharmaceutical company or Biotech company). This status is referred to as orphan designation or sometimes “orphan status”.

For a drug to qualify for an orphan designation, both the drug and the disease or condition must meet certain criteria specified in the ODA and FDA’s implementing regulations. Orphan designation qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits for qualified clinical testing.

A marketing application (when the drug is approved for safety and efficacy to be released for patients) for a prescription drug product that has received orphan designation is not subject to a prescription drug user fee unless the application includes an indication for other than the rare disease or condition for which the drug was designated.

EMA, the European Medicines Agency,  works closely on the designation and assessment of orphan medicines, in particular with FDA, sharing information on orphan medicines under their confidentiality arrangement. The two authorities have also developed common procedures for applying for orphan designation and for submitting annual reports on the status of development of designated orphan medicines.

In this matter, soon EMA will most probably follow FDA decision and grant Orphan Designation to TransCon CNP and have the process ongoing in Europe too.