EMA Recommended 17 Rare Disease Drugs in 2014

From DCAT Value Chain Insights (VCI)

By Regulatory News posted 01-12-2015 05:07


In 2014, the European Medicines Agency (EMA) recommended the highest number of orphan designated medicines for marketing authorization in a year. Out of the 82 medicines for human use recommended in 2014, 17 are intended for the treatment of a rare disease to provide therapies for patients who often have only few or no treatment options.

Among them is the first medicine for the treatment of Duchenne muscular dystrophy (PTC Therapeutics' Translarna (ataluren)) as well as the first treatment for erythropoietic protoporphyria, a rare genetic disease which causes intolerance to light (Clinuvel Pharmaceuticals' Scenesse (afamelanotide)). Last year also saw the first recommendation worldwide of a therapy based on stem cells. The orphan medicine (Chiesi Farmaceutici's Holoclar) is a treatment for limbal stem cell deficiency (LSCD), a rare eye condition that can result in blindness. Special regulatory pathways were used for these three medicines (conditional marketing authorization for Translarna and Holoclar and approval under exceptional circumstances for Scenesse). These mechanisms are in place to potentially speed up market access for medicines that fulfill unmet medical needs but for which comprehensive data cannot be provided at the time of application for a marketing authorization.

Eight new medicines for cancer were recommended for marketing authorization in 2014, of which AstraZeneca's Lynparza (olaparib), Janssen's and Pharmacyclics' Imbruvica,(ibrutinib), Roche's Gazyvaro (obinutuzumab), and Eli Lilly and Company's Cyramza (ramucirumab) target rare cancers that are difficult to treat. A targeted treatment for melanoma patients whose cancer has a specific mutation was also recommended for approval in 2014 (GlaxoSmithKline's Mekinist (trametinib).

Overall, the number of medicines containing new active substances continues to increase. One in two medicines, either orphan or non-orphan, recommended for approval in 2014 by the EMA's Committee for Medicinal Products for Human Use (CHMP) contains a substance that has never been used in medicines before. These medicines have the potential to treat diseases for which no treatments were previously available or bring added benefit to patients over existing therapies.

During the past year, the EMA provided more scientific support in the early stages of medicine development. Almost seven out of ten applicants received scientific advice from the EMA’s CHMP during the development phase of their medicine, and this figure rises to four out of five when it comes to innovative medicines. This is a significant increase compared with 2013 when only half of applicants who had a positive opinion for their medicine had received scientific advice.

Seven positive opinions were granted after an accelerated assessment in 2014: Bristol-Myers Squibb's Daklinza (daclatasvir), Gilead Sciences' Harvoni (ledipasvir and sofosbuvir), AbbVie's Exviera (dasabuvir), AbbVie's Viekirax (ombitasvir/paritaprevir/ritonavir) + Exviera (dasabuvir), Boehringer Ingelheim's Ofev (nintedanib), Janssen's Sylvant (siltuximab), and Laboratoire HRA Pharma's ketoconazole HRA. This mechanism aims to speed up the assessment of medicines that are expected to be of major benefit for public health. Four of these are for the treatment of chronic hepatitis C virus (HCV) infection. The CHMP used the accelerated assessment tool to make a new generation of medicines available to patients potentially more quickly; these treatments have high cure rates and have recently reshaped the way patients with chronic HCV infection can be treated.

In the past year, the CHMP granted a pediatric-use marketing authorization (PUMA) for Pierre Fabre Dermatologie Laboratories' Hemangiol (propranolol) for the treatment of proliferating infantile hemangioma, which are benign tumors of blood vessels. PUMAs can be granted for medicines which are already authorized, but no longer under patent or supplementary certificate protection, and that have been developed specifically for children. As an incentive to stimulate the development of existing medicines for the treatment of children, PUMA medicines are granted 10 years of market protection. For each of these medicines, EMA’s Pharmacovigilance Risk Assessment Committee  and CHMP adopted a risk management plan to ensure that safety is monitored throughout the lifecycle of the product allowing appropriate regulatory action to be taken if any new risk arises.

Of the 82 medicines recommended for approval by the EMA's CHMP in 2014, 40 were for non-orphan drugs and 17 were for orphan drugs. The EMA also recommended approval of three biosimilars, 1 pediatric drugs as previously described, and 21 generic, hybrid and informed consent drug applications.

Source: European Medicines Agency


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