2018 was very fruitful for the European Medicines Agency (EMA), which issued 84 positive opinions for human medicines, compared to only five negative ones. The new active substances passed through the caudine forks of the regulatory assessment were 42; ten applications for authorization withdrawn. Orphan drugs have been the masters even in the last year, with 21 approvals, in addition to 3 new advanced therapy products, four approvals with accelerated assessment process (150 days instead of 210) as they still respond to needs disregarded, three products approved with procedure for exceptional circumstances and one with conditional MA.

All the information are collected in the EMA overview on the authorisation and safety monitoring of medicines for human use.

The new products approved in 2018

Most of the new products are anti-cancer medicines (23 products), followed by products to fight infections (11) and neurological ones (10). Among the latter we report a product for pediatric use for the treatment of infantile spasms (West syndrome) and partial resistant epilepsy (Kigabeq, vigabatrin). Other products approved for younger patients approved in 2018 are Amglidia (glibenclamide), for the treatment of diabetes mellitus of the newborn, infants and children, and Slenyto (melatonin), aimed at children and adolescents suffering from insomnia and they are suffering from diseases of the autism spectrum.
The anti-cancer medicine Rubraca (rucaparib camsylate) has been approved with conditional authorization, with the obligation to conduct a new clinical safety and efficacy study with respect to chemotherapy to better evaluate its potential in the treatment of relapsing ovarian tumors.
All three products approved for exceptional circumstances are intended for endocrinology and are indicated, respectively, for enzyme replacement therapy of mild and moderate alpha-mannosidosis (Lamzede, velmanase alfa), for the treatment of non-neurologic manifestations of mucopolysaccharidosis VII ( Mepsevii, vestronidase alfa) and for lipodystrophy (Myalepta, metreleptina). For these products, Ema has established post-authorization obligations, which may include the creation of patient registries for long-term safety and efficacy assessment and / or the conduct of new clinical trials that deepen some aspects of treatment. An integrated immunogenicity report was also requested for Myalepta.
Another product approved as an orphan drug and recalled by Ema in its annual summary is Namuscia (mexiletine hydrochloride), dedicated to adult patients with non-dystrophic miotic disorders; it is the first product of this type approved in Europe.
Among the ATMP therapies stand out the approval of Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel), the two CAR-T therapies for the treatment of some blood cancers, and Luxturna (voretigene neparvovec-rzyl), which aims to correct the defects of RPE65 gene responsible for hereditary retinal dystrophy, a severe and rare ocular disease that can lead to blindness.
The accelerated approvals concerned the first-in-class Hemlibra product (emicizumab-kxwh), which prevents bleeding episodes in patients with haemophilia A and coagulation factor VIII inhibitors; Takhzyro (lanadelumab-flyo), based on monoclonal antibodies for the prevention of recurrences of hereditary angioedema, and two neurological products for the treatment of hereditary amyloidosis mediated by transthyretin (Onpattro, patisaran and Tegsedi, inotersen).

Lastly, the 65 new indication extensions recommended by Ema for products on the market, among which the approval of Kineret (anakinra) for the treatment of Still’s disease (a variant of rheumatoid arthritis) in adult patients and children.
The five negative opinions follow the impossibility for the CHMP committee to reach an agreement on the benefit-risk balance of the products, and have concerned EnCyzix (enclomifene, against male hypogonadism), Dexxience (betrixaban, for the prevention of venous thromboembolism ), Eladynos (abaloparatide, against osteoporosis), Alsitek (masitinib, against amyotrophic lateral sclerosis) and Exondys (eteplirsen, for Duchenne muscular dystrophy).

Finally, within the “Medicines for All” program, Ema also approved Fexinidazole Winthrop (fexinidazole) for the oral treatment of African populations suffering from sleep sickness, a tropical infectious disease with a potential risk of life caused by Trypanosoma brucei gambiense .

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