The European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) recommended 39 human-use medicines for marketing authorisation in first-half 2014, compared to 44 in the same period of 2013 and 33 in first-half 2012.
However, this year’s approvals include a number of new innovative medicines with the potential to meet unmet medical needs, treat diseases for which no treatments were previously available and bring significant added benefit to patients over existing therapies, says EMA.
They include: two cancer drugs – GlaxoSmithKline’s Mekinist (trametinib) and Roche’s Gazyvaro (obinutuzumab), Takeda’s anti-inflammatory Entyvio (vedolizumab), Bristol-Myers Squibb’s anti-infective Daklinza (daclatasvir), and two products for the treatment of rare hereditary conditions – PTC Therapeutics’ Translarna (ataluren) and Janssen’s Sylvant (siltuximab).
The number of products recommended for approval via the European Union (EU) centralised procedure, based on generic or informed consent applications, also fell, to six compared with 13 in first-half 2013.
The number of new medicines for rare diseases is steadily increasing, providing treatments for patients who often have only few or no options, the Agency notes. In first-half 2014, eight such treatments received a CHMP recommendation, although they include three for which the panel recommended conditional approval but whose applications were withdrawn by the sponsor prior to a final decision by the European Commission.
Conditional approval – a mechanism used to provide early patient access to medicines that fulfil unmet medical needs or address life-threatening diseases – was also used by the CHMP to recommend Translarna, the first treatment for Duchenne muscular dystrophy, a life-threatening condition.
The Committee granted two positive opinions after an accelerated assessment for Sylvant and Daklinza; this mechanism aims to speed up the assessment of medicines that are expected to be of major public health interest, particularly from the point of view of therapeutic innovation, it says.
The CHMP also gave an opinion on the use of a new combination product – ledipasvir and sofosbuvir – in the treatment of hepatitis C virus (HCV) in a compassionate-use programme.
In first-half 2014, more than two out of three applicants received scientific advice from the Committee during their medicine’s development phase, while for innovative medicines four in five applicants received such advice. This is a significant increase compared with first-half 2013, when one in two applicants received scientific advice, and this mirrors the growing number of scientific advice requests which EMA is now receiving, it says.
