Five therapies have been backed for approval in the European Union, including two orphan medicines addressing rare conditions.
First up, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) endorsed AMMTeK’s Amglidia (glibenclamide) for the treatment of neonatal diabetes mellitus in newborns, infants and children.
Neonatal diabetes is an extremely rare form of diabetes diagnosed in the first six months of life. The condition is life-threatening and debilitating because of the symptoms caused by high blood sugar levels and the risk of ketoacidosis.
Amglidia, a new oral formulation of diabetes drug glibenclamide developed specifically for paediatric use, will be the first medicine approved in the region to treat the condition if the EMA follows the CHMP’s recommendation. The drug works by facilitating the release of insulin into the bloodstream.
The other orphan medicine in the batch is Pfizer’s Mylotarg (gemtuzumab ozogamicin), which received a positive opinion for the treatment of acute myeloid leukaemia in patients aged 15 years and above.
Biocryst UK’s Alpivab (peramivir) won CHMP backing as a treatment for uncomplicated influenza. The drug works by inhibiting virus neuraminidase, an enzyme important for viral entry into uninfected cells and release and spread of new virus once cells have been infected.
Elsewhere, the CHMP granted a positive opinion for two informed consent applications, which are part based data from the dossier of a previously authourised medicine.
Chiesi’s CHF 5993 Chiesi Farmaceutici S.p.A. (beclometasone dipropionate anhydrous/formoterol fumarate dihydrate/glycopyrronium) and Trydonis (beclometasone dipropionate anhydrous/formoterol fumarate dihydrate/glycopyrronium) were both backed as treatments for adults with moderate to severe chronic obstructive pulmonary disease.
On the down side, the Committee did not back approval of Puma Biotechnology’s Nerlynx (neratinib) for breast cancer, or extending the scope of Pfizer’s Sutent (sunitinib) to include the delay or prevention of the return of kidney cancer in patients who have had surgery and are at high risk of their cancer coming back.
While trial data showed that a greater proportion of women given Nerlynx lived for two years without their disease coming back versus those given placebo (around 94 percent versus 92 percent, respectively), “it is uncertain that this difference in benefit would be seen in clinical practice,” the agency noted.
Also, the drug causes side effects in the digestive system, particularly diarrhoea, which affected most patients and might be difficult to manage, throwing a further shadow of doubt over its benefit-risk profile, it added.
For Sutent, the CHMP said the evidence that it delays the return of the cancer was not convincing, even when looking at data from those patients considered at highest risk.
