AZ's gMG drug Klygefa nears EU approval

AstraZeneca's rare disease unit Alexion has claimed a positive opinion from the EMA's human medicines committee for generalised myasthenia gravis (gMG) therapy Klygefa.

The recommendation by the CHMP sets up an EU approval in the coming weeks for Klygefa (gefurulimab), a dual-binding nanobody C5 inhibitor that extends Alexion's presence in the C5 inhibitor class. The drug is already approved for marketing in Japan and is being prepared for filing in other markets, including the US.

The company already sells two drugs in the C5 inhibitor class – Soliris (eculizumab) and Ultomiris (ravulizumab) – which are given as intravenous infusions, but Klygefa can be administered as a subcutaneous injection by gMG patients themselves. Combined sales of Soliris and Ultomiris were around $6.5 billion last year.

If approved, Klygefa would offer once-weekly dosing at home, and would provide another weekly subcutaneous treatment option for gMG patients in the EU alongside Argenx's Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase) and UCB's Rystiggo (rozanolixizumab) – both FcRn blockers. Some analysts have suggested the drug could eventually reach $1 billion or more in peak sales.

Other CHMP decisions

Along with a positive recommendation for Novo's haemophilia A therapy Frehemgo (see our article here), the CHMP also backed half a dozen other novel medicines, including:

SFL Pharma's ALK inhibitor Gevalka (ensartinib) for the first-line treatment of adult patients with ALK-positive advanced non-small cell lung cancer (NSCLC), and PARP inhibitor Sepalna (senaparib) as a maintenance treatment for patients with advanced epithelial high-grade ovarian, fallopian tube or primary peritoneal cancer who are in complete or partial response following first-line platinum-based chemotherapy;

Corcept Therapeutics' glucocorticoid receptor (GR) antagonist Lifyorli (relacorilant), for the treatment of platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer, in combination with paclitaxel, after one to three prior systemic treatment regimens;

Incyte's selective JAK1 inhibitor Povofortay (povorcitinib) for moderate to severe hidradenitis suppurativa – sometimes known as acne inversa – in adults who have responded inadequately to, or who are intolerant or contraindicated to an anti-TNF medicine;

Zealand Pharma's GLP-2 analogue Zeydovio (glepaglutide) for the treatment of short bowel syndrome in patients who have been stable after a period of intestinal adaptation after surgery; and

Sanofi's VaxRabeo, a vaccine for pre-exposure and post-exposure prophylaxis against rabies – a viral infection that is virtually 100% fatal in humans once symptoms appear – in individuals of all age groups. Some rabies vaccines have strict age restrictions, so VaxRabeo has the potential to simplify emergency treatment.