PTC Therapeutics, Inc. PTCT announced that it received a $20-million milestone payment from Roche Holding AG RHHBY under its license and collaboration agreement for its first commercial sale of Evrysdi (risdiplam) in the United States. Shares of PTC Therapeutics have lost 0.3% so far this year compared with the industry’s decline of 6.3%.
Evrysdi was approved by the FDA on Aug 7 for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older. With the approval, the drug became the first at-home administered liquid medicine for people with SMA. SMA is a hereditary disease that causes weakness and muscle wasting because patients lose lower motor neurons (nerve cells) that control movement. The marketing authorization application (MAA) for Evrysdi for the treatment of SMA was accepted by the European Medicines Agency on Aug 18.
Evrysdi is a product of the SMA collaboration between PTC, the SMA Foundation and Roche. Roche leads the development of the drug.
However, competition is stiff in the SMA market from the likes of Biogen’s BIIB Spinraza and Novartis’ NVS gene therapy, Zolgensma.
PTC Therapeutics has a pipeline of gene therapy product candidates for rare monogenic diseases that affect the central nervous system (“CNS”) including PTC-AADC for the treatment of Aromatic L-Amino Acid Decarboxylase, (AADC) deficiency, a rare CNS disorder arising from reductions in the enzyme AADC thatresults from mutations in the dopa decarboxylase gene.
PTC Therapeutics currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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