APLT - Applied Therapeutics govorestat gets orphan drug tag in EU for muscle disorder

2023-05-25 07:49:29 ET

The European Medicines Agency (EMA) granted orphan medicinal product designation to Applied Therapeutics' ( NASDAQ: APLT ) drug AT-007 (govorestat) to treat  Sorbitol Dehydrogenase (SORD) Deficiency.

SORD Deficiency is a rare hereditary disorder which affects peripheral nerves and motor neurons causing muscle weakness. The disorder mainly affects the lower limbs leading to difficulty in walking.

"We are pleased that the EMA has recognized the high unmet medical need in SORD Deficiency, and the benefit of govorestat treatment in reducing toxic sorbitol levels in patients with SORD Deficiency as well as preventing disease progression in the animal model of disease," said Applied's Founder and CEO Shoshana Shendelman.

The orphan drug status in the EU is aimed at therapies treating or preventing diseases which affect fewer than five in 10,000 people in the EU. The designation provides incentives, including 10 years of market exclusivity for the drug, if approved.

In addition, the company also announced that new data was published in the Journal of Clinical Investigation on govorestat treatment in models of SORD Deficiency.

Govorestat significantly reduced sorbitol levels in patient-derived fibroblasts, patient iPSC-derived motor neurons and SORD-deficient drosophila, according to Applied.

APLT added that govorestat therapy in the SORD drosophila model mitigated synaptic degeneration and improved synaptic transduction, locomotor activity, and mitochondrial function. The drug also reduced ROS accumulation in patient derived cells and drosophila CNS and muscle.

APLT +5% to $1.47 premarket May 25

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