MHRA designates VTS-270 as Promising Innovative Medicine for children with Niemann-Pick Type C1 disease

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The Medicines and Healthcare Products Regulatory Agency (MHRA) has granted a Promising Innovative Medicine (PIM) designation for VTS-270, an investigational drug for children with Niemann-Pick Type C1 disease.

Vtesse, a company that develops drugs for patients with extremely rare, life-threatening diseases, is behind the drug.

The PIM designation is the first step to inclusion in the Early Access to Medicines Scheme (EAMS), which provides patients with faster access to innovative medicines.

Dr Ben Machielse, President and Chief Executive Officer of Vtesse, said: “It takes considerable collaboration and dedication to advance the clinical study and regulatory processes for drug development in the rare disease space. Above all, we thank all the parents who have supported the development of VTS-270.”

VTS-270 was also granted U.S. FDA Breakthrough Therapy Designation earlier this year.