Orphan drugs lead CHMP recommendations

The first medicine developed to treat a rare disease where people are intolerant to sunlight was among six new drugs recommended for use in Europe today.

Scenesse (afamelanotide), developed by Australian company Clinuvel Pharmaceuticals, was one of two orphan medicines for rare diseases recommended in the latest round of opinions from the Committee for Medicinal Products for Human Use (CHMP).

The CHMP, part of the European Medicines Agency (EMA), backed the use of Scenesse for treatment of erythropoietic protoporphyria (EPP), a rare genetic disease that leads to severe burns, pain, swelling and scarring when a person is exposed to sunlight.

Scenesse combats the disease by stimulating the production of a pigment called eumelanin, which protects the skin against sunlight.

The recommendation, which is based on exceptional circumstances due to the rarity of the disease and the difficulty of obtaining comprehensive data, puts the drug on course to be the first approved treatment for EPP in the EU.

Significantly for the EMA, the recommendation was reached after patients were invited to share their experiences of living with the condition to the CHMP. This was the first time that patients have been involved in the CHMP assessment of a medicine, although the EMA has plans to further embed the patient viewpoint in its decision making process

AstraZeneca's olaparib

Scenesse wasn't the only orphan drug for a rare disease to be recommended by the CHMP as AstraZeneca (AZ) received backing for the use of Lynparza (olaparib) in the treatment of a subtype of ovarian cancer where women have a mutation in the BRCA gene.

Lynparza is among the first in the new PARP (poly ADP-ribose polymerase) inhibitor class to come through development and has demosnated impressive results in BRCA-mutated ovarian cancer. However, approval for the drug has stalled in the US with an FDA panel requesting more data.

More recommendations

Four more new treatments were recommended by the CHMP, including Pfizer's Duavive (estrogens conjugated/bazedoxifene), a replacement therapy for postmenopausal women with oestrogen deficiency.

Baxter received a positive opinion for Rixubis (nonacog gamma) in the treatment and prevention of bleeding in patients with haemophilia B.

The CHMP also granted positive opinions for two informed consent applications: Lilly's Duloxetine Lilly (duloxetine) for the treatment of major depressive disorder, diabetic peripheral neuropathic pain and generalised anxiety disorder, and Janssen's Paliperidone Janssen (paliperidone) for the treatment of schizophrenia.