Boston, MA 05/23/2014 (wallstreetpr) – Biopharmaceutical Company, PTC Therapeutics, Inc. (NASDAQ: PTCT), is surging by as much as 72.43% after the Committee for Medicinal Products for Human CHMP use adopted a positive opinion regarding its application for conditional marketing authorization of Ataluren.
European Commission Verdict
Attention now shifts to the European Commission, expected to give a decision as to whether PTC Therapeutics, Inc. (NASDAQ:PTCT) will enjoy conditional marketing authorization rights. CHMP opinion is expected to have a huge impact on the commission decision. The commission is expected to give its verdict in three months.
Approval will essentially give the company the right to distribute product in the 28 countries that are members of the European Union. PTC requested for re-examination after it failed to persuade the commission in January consequently receiving negative opinion. Approval of the drug by CHMP is a major milestone for the company that continues to work closely with the community.
Positive CF Trials
Approval by CHMP follows the company reporting impressive results for clinical trials of Ataluren on the treatment of Cystic fibrosis. The results showed positive trends in both the primary end point and the secondary outcome.
Data from the trials essentially shows that Ataluren can result in meaningful improvements compared to placebo. Patients on whom tests were carried by the use of Ataluren showed fewer pulmonary exacerbations with meaningful stabilization in FEV1 results.
Nonsense mutations CF patients never produce meaningful functional CFTR protein thus the reason behind their severe form of severe Cystic Fibrosis. Current treatments in the market solely focus on reducing various forms of infections and alleviating symptoms. Ataluren on its part targets the underlying cause of the condition according to Michael Konstan a lead study investigator.
The trials on Ataluren also showed impressive safety results that were tolerable. The study points to potential first in class treatment for nmCF patients not using chronic inhaled tobramycin. Ataluren and placebo registered similar results through week 48 with slight changes in the occurrence of creatinine elevations.
Serious adverse events involved CF Pulmonary exacerbations that were not related to Ataluren treatment. During the trials, 8 patients under Ataluren and 3 under placebo withdrew due to adverse events.
