CHMP backs Roche's Enspryng for rare nerve disease NMOSD

Roche Rituxan approval

Roche's Enspryng has been recommended for approval in the EU for treating neuromyelitis optica spectrum disorder (NMOSD), extending the treatment options for people with the life-threatening rare disease. 

The CHMP has backed the drug in patients aged 12 or more with NMOSD that tests positive for aquaporin-4 (AQP4) antibodies, a biomarker seen in around 80% of NMOSD cases. Enspryng was approved for the same indication in the US last year.

NMOSD is a lifelong and debilitating autoimmune disorder of the central nervous system, often misdiagnosed as multiple sclerosis, that causes damages to the optic nerves and spinal cord, leading to blindness, muscle weakness and paralysis. It affects roughly one to two people per 100,000 in the EU.

IL-6 inhibitor Enspryng isn't the first drug to treat NMOSD to get a green light from the CHMP, as Alexion's complement C5 blocker Soliris (eculizumab) has been approved for the disease in the EU since 2019. However, according to Roche is the only therapy that can be used in adolescents over 12 as well as adults. Alexion has phase 3 trials on the go in children and adolescents with NMOSD.

The Swiss pharma group also says its product is the only subcutaneous treatment option for NMOSD that can be administered at home every four weeks, after an initial three injections given two weeks apart. Soliris is given by intravenous infusion every two weeks, after an initial period of weekly dosing.

Another NMOSD therapy, Horizon Pharma/Viela Bio's Uplizna (inebilizumab), has been approved in the US but isn't yet available in Europe. It too is administered by intravenous infusion.

Meanwhile, Alexion's follow-up to Soliris – Ultomiris (ravulizumab) – is also in late-stage clinical development as an infusion for NMOSD, although the drugmaker is also working on a subcutaneous formulation of the antibody. Also back in early-stage development is an oral therapy based on cladribine developed by Swiss biotech Chord Therapeutics.

Enspryng's approval based on two clinical trials which showed that the rate of relapse with Enspryng was a quarter of that in a placebo group, both given on top of other immunosuppressive drugs.

Roche's drug was one of several new medicines recommended for approval at the CHMP's April meeting.

Regeneron's first-in-class ANGPTL3 inhibitor Evkeeza (evinacumab) got a positive opinion for homozygous familial hypercholesterolemia (HoFH), a genetic form of high cholesterol, in patients aged 12 and over, and the committee also backed AstraZeneca's MEK 1/2 inhibitor Koselugo (selumetinib) for children with neurofibromatosis type 1 (NF1) plexiform neurofibromas (PN).

Leo Pharma's IL-13 inhibitor Adtralza (tralokinumab) got the go-ahead as a treatment for adults with moderate to severe atopic dermatitis, as did Celgene’s Onureg (azacitidine) as a maintenance therapy for patients with acute myeloid leukaemia (AML).

Other highlights included an extension of the Indications for AbbVie's Venclyxto (venetoclax) as a first-line treatment – in combination with azacytidine or decitabine – in newly-diagnosed AML patients ineligible for intensive chemotherapy.

AZ also got the go-ahead for Tagrisso (osimertinib) for the adjuvant treatment after surgery of patients with early-stage EGFR-positive non-small cell lung cancer (NSCLC).