December 2020 • PharmaTimes Magazine • 8-9
// MEDICINE //
Six meds near EU approval
Six medicines have taken a leap towards approval having won the backing of the European Medicines Agency's human medicines committee (CHMP).
Roche's Phesgo, a fixed dose combination of pertuzumab and trastuzumab, was endorsed as a treatment for early and metastatic breast cancer. According to the Committee, the treatment offers patients “a less invasive and faster administration as a single product, compared to the current administration of the approved intravenous pertuzumab and subcutaneous trastuzumab formulations”.
A positive opinion was also adopted for the Swiss drug giant's Xofluza (baloxavir marboxil), for the treatment and post-exposure prophylaxis of uncomplicated influenza.
Aerie Pharmaceuticals Ireland's Roclanda (latanoprost/netarsudil) was backed for the reduction of elevated intraocular pressure (IOP) in adult patients with primary open-angle glaucoma or ocular hypertension for whom monotherapy with a prostaglandin or netarsudil provides insufficient IOP reduction.
Samsung Bioepis' biosimilar Onbevzi (bevacizumab) won CHMP backing for the treatment of carcinoma of the colon or rectum, breast cancer, non-small cell lung cancer, renal cell cancer, epithelial ovarian, fallopian tube or primary peritoneal cancer and carcinoma of the cervix. The Committee noted that Onbevzi has comparable quality, safety and efficacy to Avastin (bevacizumab), which was authorised in the EU in January 2005.
The CHMP also recommended approving the marketing authorisation for Stemline Therapeutics' Elzonris (tagraxofusp), for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare and aggressive type of acute myeloid leukaemia (blood cancer).
The Committee had initially refused the application in July 2020 for Elzonris to be used for the treatment of BPDCN regardless of whether patients had been previously treated with other medicines or not. After re-examination, it recommended approval under exceptional circumstances but for a restricted indication for first-line treatment.
On the downside, the Committee stuck with a negative recommendation on Swedish Orphan Biovitrum's Gamifant (emapalumab). Having re-examined the application, the CHMP felt that safety concerns had been addressed, but that questions remain over efficacy. The drug was intended for the treatment of primary haemophagocytic lymphohistiocytosis (a genetic disease characterised by an overactive immune system) in children under 18 years of age.
NICE backs Lilly’s Emgality
The UK’s National Institute for Health and Care Excellence (NICE) has recommended NHS funding of Eli Lilly’s Emgality (galcanezumab) as a preventative treatment for migraine in adults with episodic and chronic migraine.
Emgality, a once-monthly subcutaneous injection, binds to the calcitonin gene-related peptide (CGRP), which is believed to play a role in migraine attacks, and is the first in the class to be endorsed by NICE in this setting.
The drug's use on the NHS is being funded as an option for preventing migraine in adults if: they have four or more migraine days a month, at least three preventive drug treatments have failed and the company provides it according to the commercial arrangement.
The decision is based on clinical trials comparing Emgality with placebo, in which Lilly's drug halved the number of monthly migraine headache days for up to 40% of adults with migraine who had previously tried three or more preventative medicines.
“This is wonderful news for people living with this very painful and debilitating neurological condition. Both chronic and episodic migraine patients across England and Wales will be able to access an effective drug on the NHS that has been specifically designed to prevent migraine,” commented Gemma Jolly, Migraine Trust, head of Information and Support. “Migraine significantly impacts the lives of those who live with it and access to this important new medication will be life-changing for many.”
Around 144,000 people with episodic migraine and 59,000 people with chronic migraine may be eligible for treatment with Emgality.
UK HAE patients early access to berotralstat
Patients with hereditary angioedema (HAE) living in the UK will get early access to BioCryst's berotralstat before the drug is officially approved for this indication by the European Commission.
The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted oral, once-daily berotralstat a positive scientific opinion through the Early Access to Medicines Scheme (EAMS), thus allowing patients aged 12 years and older to receive treatment with the drug for the routine prevention of recurrent attacks of HAE.
HAE is a rare, potentially life-threatening, genetic disorder causing periodic episodes of acute swelling of the skin, pharynx, larynx, gastrointestinal tract, genitals and/or extremities.
“There are many patients in the UK that don’t have a realistic option for effective HAE prophylaxis. The addition of berotralstat through the EAMS will bring a much needed option for HAE patients suffering with this debilitating disease,” said Dr Sorena Kiani, consultant immunologist at Royal London Hospital, London.
“HAE patients around the world are waiting for an oral, once-daily therapy to prevent attacks and reduce their burden of therapy. With this decision by the MHRA, the wait for many HAE patients in the UK can end sooner,” said Jon Stonehouse, chief executive officer of BioCryst.
The European Medicines Agency (EMA) is reviewing the marketing authorisation application (MAA) for berotralstat under the centralised procedure; an opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected by the end of March 2021.