CHMP expected to reject Santhera's FA drug

Santhera Pharmaceuticals, a Swiss specialty drugmaker focused on neuromuscular diseases, says that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMEA) has informally advised that it would not support a positive opinion for the Marketing Authorization Application for SNT-MC17/idebenone to treat patients with Friedreich's Ataxia.

According to the information received, Santhera's ongoing Phase III trials prevented the CHMP from reviewing the firm's application under the EMEA Guideline on Clinical Trials in Small Populations as requested by Santhera in its MAA filing. The Committee believes it cannot approve the drug before data from at least one of the advanced Phase III programs in Europe and the USA are submitted for review. The drug was approved in Canada last week (Marketletter July 28).

In clinical studies submitted to the CHMP as part of the approval process, idebenone showed statistically and clinically relevant improvements in FA patients, as measured by Activities of Daily Living scores as well as in cardiac and neurological functions. The CHMP Joint Assessment Report supports idebenone as sufficient to potentially meet its criteria for approval. However, the Committee concluded that the Swiss firm had not submitted a sufficient set of data demonstrating a clear positive risk versus benefit balance for approval under standard clinical review guidelines.