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Regulatory Focus™ > News Articles > EMA Recommends Vertex's Cystic Fibrosis Drug Kalydeco

EMA Recommends Vertex's Cystic Fibrosis Drug Kalydeco

Posted 25 May 2012 | By Alexander Gaffney, RAC 

The European Medicines Agency (EMA) has recommended Vertex Pharmaceuticals' cystic fibrosis (CF) drug Kalydeco (ivacaftor), moving the drug one step closer to becoming the drug the first in Europe for the treatment of CF in patients with a specific gene mutation.

The drug received the Committee for Medicinal Products for Human Use's (CHMP) recommendation on 25 May after being reviewed under a 150-day accelerated approval program. EMA notes Kalydeco is the eighth product to be approved through its accelerated approval pathway.

The drug offers an "innovative therapeutic approach" to treating CF in that it treats the "underlying mechanism of the disease" instead of just its symptoms, explained EMA in a statement explaining its approval of the drug.

The drug now moves to the European Commission, which will adopt a decision on the drug.

Kalydeco was approved by the US Food and Drug Administration (FDA) on 31 January 2012.

Read more:

EMA - European Medicines Agency recommends first-in-class medicine for treatment of cystic fibrosis

Vertex - Vertex Receives European CHMP Positive Opinion for KALYDECO™ (ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis

Related: EC - Commissioner Dalli delivers speech on rare diseases and orphan products

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