Today, 11th July, Dynacure announced that they have been granted Orphan Drug Designation from the European Medicines Agency (EMA) for DYN101, Dynacure’s antisense treatment for centronuclear myopathy. DYN101 is anticipated to begin clinical trials in Europe in the second half of 2019.
“With Orphan Drug Designation in the EU, we have achieved a significant regulatory milestone in our development plans for DYN101 and we look forward to working with the EMA as we progress,” said Stephane van Rooijen (M.D. MBA), Chief Executive Officer of Dynacure. “In the second half of 2019, we expect to initiate our first-in-human study, which brings us one step closer to delivering the first disease-modifying therapy designed to treat several forms of CNM.”
Leen Thielemans, Chief Development Officer of Dynacure, added, “Orphan drug status is granted to investigational therapies that are expected to make a significant impact on patient care and we believe DYN101 holds great promise to treat a broad range of centronuclear and myotubular myopathies. The formal designation has been granted based on compelling preclinical data in several forms of CNM. As we prepare to initiate the UNITE-CNM clinical study, we also intend to expand the use of DYN101 to explore additional indications where the overexpression of DNM2 is a disease-driving factor.”
Orphan Drug Designation brings many advantages both to the company developing the drug, and to the patient community:
- the European Medicines Agency provides a special form of scientific advice called ‘Protocol Assistance’, which helps the drug developer to answer critical questions about the drug development, the design of the clinical trial and particularly the criteria for getting the drug authorised for use in patients,
- reduced fees from EMA regulators for scientific advice, thereby encouraging these companies to talk to the regulators
- the ability to apply for marketing authorisation (ie. the drug approved to be offered to patients) centrally across all of the countries in the EU. This allows companies to make a single application to the European Medicines Agency, resulting in a single opinion and a single decision from the European Commission, valid in all EU Member States
- eligibility for ‘conditional’ marketing authorisation. This is where, in some cases an unauthorised medicine may be allowed to be administered to patients outside a clinical study, on compassionate grounds.
- 10 years of market exclusivity to provide the drug (plus 2 more years for drugs for children), thereby encouraging companies to want to develop drugs for rare diseases.
Orphan Drug Designation can be applied for any treatment for a condition that:
- must be intended for the treatment of a disease that is life-threatening or chronically debilitating;
- the prevalence of the condition in the EU must not be more than 5 in 10,000;
- no satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorised,
- or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.