Seven therapies backed for EU approval

Seven therapies have been put forward for approval in the European Union, bringing closer new options for a range of conditions including Crohn’s disease, diabetes and cancer.

Included in the latest pack of Committee for Medicinal Products for Human Use (CHMP) recommendations is Tigenix’ Alofisel (darvadstrocel), a new advanced therapy medicinal product (ATMP) for the treatment of complex perianal fistulas in patients with Crohn’s disease.

Perianal fistulas, a common complication of Crohn’s disease, occur when an abnormal passageway develops between the rectum and the outside of the body, potentially leading to incontinence and sepsis. Complex fistulas, which are rare, are more treatment resistant than simple fistulas.

Alofisel contains expanded adipose stem cells that interrupt proliferation of lymphocytes and reduce the release of pro-inflammatory cytokines at inflammation sites, which lessens inflammation and potentially allows the tissues around the fistula tract to heal.

In clinical trials, after 24 weeks of treatment half of patients treated with Alofisel were found to be in remission, compared to a third of the patients given aplacebo.

Elsewhere, the Committee also recommended granting a conditional marketing authorisation for Kyowa Kirin’s Crysvita (burosumab), as a treatment for X-linked hypophosphataemia with radiographic evidence of bone disease in children and adolescents with growing skeletons.

There is currently no authorised medicine available to treat this rare, serious, chronic and debilitating disease, an inherited disorder characterised by low levels of phosphate in the blood, leading to soft, weak bones.

A paediatric-use marketing authorisation (PUMA) for Diurnal’s Alkindi (hydrocortisone) was backed for the treatment of primary adrenal insufficiency, a rare hormonal disorder in infants, children and adolescents.

Children are currently treated by crushing tablets of an adult formulation of hydrocortisone prepared by a pharmacist, leading to a risk of under- or over-dosing when parents try to administer the bitter-tasting crushed tablet as best as they can. Alkindi’s formulation is meant to allow a more accurate dosing of hydrocortisone in children, with a better masking of the bitter taste, which could be helpful in particular in younger children, the CHMP noted.

Novo Nordisk’s Ozempic (semaglutide) won a positive opinion for the treatment of type II diabetes.

The drug, which is approved in the US, is a glucagon-like peptide 1 (GLP-1) receptor agonist with once-weekly dosing, which induced clinically meaningful and statistically significant reductions in HbA1c compared with placebo, sitagliptin, exenatide extended-release and insulin glargine U100 in clinical trials.

Also put forward for approval were Celltrion Healthcare Hungary’s trastuzumab biosimilar Herzuma, for the treatment of breast and gastric cancer, and the generics Anagrelide Mylan, for the reduction of elevated platelet counts in at risk essential thrombocythaemia patients, and KRKA/Novo mesto’s Efavirenz/Emtricitabine/Tenofovir disoproxil Krka, for the treatment of HIV infection.

On the downside, the CHMP adopted a negative opinion for Pharma Mar’s Aplidin (plitidepsin) as a treatment of multiple myeloma.