On 3 July 2012, the Committee for Medicinal Products for Human Use (CHMP) recommended the refusal of a marketing authorisation for the medicinal product Elelyso (manufactured by Protalix Biotherapeutics, Israel and marketed outside of Israel by Pfizer Ltd), intended for the treatment of type 1 Gaucher disease.
The CHMP noted that the main study showed that Elelyso led to clinically relevant reductions in the size of the spleen as well as the liver. There were also improvements in haemoglobin levels and blood platelet counts. The side effects seen with Elelyso appear to be similar to those of other enzyme replacement therapies. The CHMP therefore concluded that the benefits of the medicine outweighed its risk in the treatment of type 1 Gaucher disease.
However, the CHMP also concluded that the medicine cannot be granted marketing authorisation in the EU because of the ten-year market exclusivity that had been granted for Vpriv, which was authorised in August 2010 for the same condition. Market exclusivity for orphan medicines is given as an incentive for companies to develop medicines for rare diseases, which may otherwise not be developed due to the high costs and small patient populations. The exclusivity means that another medicine cannot be authorised for the same condition if it is similar to the medicine already authorised. In this case, the CHMP concluded that Elelyso is similar to Vpriv (made by Shire Human Genetics), as they are both enzyme replacement therapies that work in the same way.
The CHMP also considered the legal exemptions that could have allowed Elelyso to be authorised in spite of Vpriv’s market exclusivity. The possible exemptions related to whether Elelyso was clinically superior to Vpriv and whether there were supply problems with Vpriv. However, the CHMP concluded that there is no good evidence that Elelyso would offer patients any important advantages over Vpriv or that Vpriv is in short supply. The Committee therefore recommended the refusal of the marketing authorisation.
The European Gaucher Alliance (EGA) wrote to the Chair of the CHMP and the European Commission to highlight the disappointment and dismay of the Gaucher patients in Europe by this recommendation. Stressing that if this recommendation is accepted by the Commissioners that Gaucher patients within the European Union will be disadvantaged when compared to patients in the United States where the FDA has given authorisation to Elelyso as their doctors will not be able to offer the same range of alternative Enzyme replacement therapies.
Despite all the lobbying of the EGA and the individual patient organisations in European, Pfizer were informed by the European Commission on Friday 2nd November 2012 that they were to follow the CHMP’s recommendation and not authorise Elelyso for marketing.
Gaucher patients in Europe currently participating in an Elelyso clinical trial will continue as planned in the protocol while other Elelyso patients should discuss their future treatment opportunities with their treating physicians.