Curlim Receives EMA Orphan Drug Designation for CLM001

We are pleased to announce that the European Commission, following a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA), has granted CLM001 orphan drug designation for the treatment of Charcot-Marie-Tooth disease (CMT).

This official recognition confirms the significant therapeutic potential of CLM001 for a rare, chronic, and severely disabling condition, for which no curative option is currently available in the European Union.

CLM001: A breakthrough therapeutic innovation

Preclinical data submitted to the EMA show that, in several relevant animal models of CMT1A (one of the most common forms of the disease) , CLM001:

significantly improves nerve conduction
Improves movement and coordination
demonstrates strong therapeutic potential for future clinical evaluation

These robust results were key in the COMP’s decision, which concluded that CLM001 meets all the criteria set out in Regulation (EC) No 141/2000 for orphan drug designation:

A rare and seriously debilitating disease
Absence of satisfactory treatment
Established medical plausibility supported by solid non-clinical data

A rare disease with urgent medical needs

CMT affects approximately 3.1 people per 10,000 in Europe.
This progressive hereditary neuropathy gradually damages the peripheral nerves, leading to:

muscle weakness
walking difficulties
chronic pain
and a substantial loss in quality of life.

Despite its severe and irreversible impact, there is currently no treatment to slow down or reverse its progression. This makes innovation essential to meet this urgent medical need.

A key milestone towards clinical trials

This designation grants Curlim several strategic regulatory advantages in Europe, including:

Facilitated clinical development through EMA scientific advice
10 years of market exclusivity after marketing authorization
Priority access to innovation support measures (fee reductions, pediatric investigation plan, etc.)

To consult the official entry in the EU Community Register of Orphan Medicinal Products (EU/3/25/3092) :
View the register