AMT Receives EMEA Orphan Drug Designation for Acute Intermittent...( AMSTERDAM May 28 /- Amsterdam Mol...),Health

AMSTERDAM, May 28 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that the European Medicines Agency has granted Orphan Drug Designation to AMT's gene therapy product AMT-021 for the treatment of acute intermittent porphyria (AIP).

Orphan Drug Designation for AIP entitles AMT to ten year market exclusivity in Europe following marketing approval for AMT-021 if this product candidate is the first new drug with a major medical benefit receiving marketing approval for the European Union. The designation also provides for special benefits, including research support, eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees during development or at the time of application for marketing approval.

"We are proud to have received this Orphan Drug Designation for the treatment of acute intermittent porphyria," said Sander van Deventer, Chief Executive Officer of AMT. "This designation is an important step in the development of a treatment for this seriously debilitating and potentially lethal disease."

About Acute Intermittent Porphyria

Acute intermittent porphyria is a rare genetic disease in which mutations in the PBGD gene result in insufficient activity of a protein necessary for the synthesis of heme. This leads to an accumulation of toxic intermediate metabolites resulting in a wide variety of problems including acute, severe abdominal pains, psychiatric, neurologic illnesses, and muscular weakness. Long-term consequences may be irreversible nerve damage, liver cancer and kidney failure. Acute porphyric attacks can be life-threatening.

AIP affects 1 per 10.000 people in the European Union.

Currently available therapies do not prevent the symptoms and consequences of acute porphyric attacks. AMT has demonstrated that its prod
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