Approvals

The National Institute for Health and Care Excellence (NICE) have recommended adult patients in England or Wales with moderately or severely active ulcerative colitis (UC) who have not responded well to or cannot tolerate conventional or biological therapy including a tumour necrosis factor (TNF) alpha inhibitor the option of skyrizi (risankizumab).

UC patients who lost or have had inadequate response, or were intolerant to, conventional and/or biologic therapy also had risankizumab approved by the Medicines and Healthcare Products Regulatory Agency (MHRA) a week prior.

The recommendation from NICE is based on results from two phase 3 clinical trials. The introduction trial, INSPIRE, evaluated intravenous risankizumab at a dose of 1200mg, administered as an introduction dose to adult patients with moderately to severely active UC at zero, four and finally eight weeks. Patients who responded to the introduction treatment were re-randomised for the second trial, COMMAND. In this trial, patients received a subcutaneous (SC) injection of risankizumab as a maintenance dose, at a dosage of either 180mg or 360mg. The maintenance doses were administered over a period of 52 weeks.

Medical director of AbbVie UK Rachael Millward stated: “AbbVie is committed to addressing the unmet needs of patients living with IBD. We are delighted that following the NICE recommendation, risankizumab will be made available for suitable UC patients."

The safety profile of risankizumab in both trials was consistent with the safety profile observed in previous trials across other indications, with no new safety risks observed.

UC has a higher prevalence than Crohn’s disease (CD) or unclassified inflammatory bowel disease (IBD) in the UK, according to a report by Crohn's & Colitis UK, with 296,000 people estimated to be living with UC today in the UK, and at least one in every 227 people diagnosed. The severity of symptoms and unpredictability of flares can lead to a substantial burden and often cause disability for those living with the disease. More than one in three people with CD or UC identify as disabled.

Dr Gareth Parkes, consultant gastroenterologist, Barts Health NHS Trust UK stated: “The unpredictable and potentially debilitating nature of UC can cause significant emotional stress for patients. With UC becoming increasingly more common, I’m pleased that we now have another treatment option to offer eligible patients in England and Wales, which may help them to gain long-term control of their condition.”

Zevra Therapeutics has announced that the US Food and Drug Administration’s (FDA) Genetic Metabolic Diseases Advisory Committee (GeMDAC) has voted in favour of data supporting its Niemann-Pick disease type C (NPC) treatment.

The vote comes three years after arimoclomal – owned by Orphazyme at the time – was initially rejected by the FDA, who requested an additional clinical trial was run to provide more robust data. The GeMDAC decision took into consideration the original 50-subject phase 2/3 Orphazyme trial, as well as results from the four-year open label extension, eight lab studies, and rea l-world data from early access programmes.

Neil F McFarlane, president and chief executive officer of Zevra, stated: “We are extremely pleased with the committee’s recognition of the benefits of arimoclomol for people living with NPC. Based on the totality of the clinical data, including data from the pivotal trial, the long-term data from the arimoclomol open label extension study, and data from our expanded access programmes, we remain confident in the clinical benefit offered by arimoclomol as a treatment for NPC, and are optimistic about its continued path to approval.”

Arimoclomol, an orally delivered, investigational drug candidate, has previously been granted Orphan Drug, Fast Track, Breakthrough Therapy and Rare Paediatric Disease designations by the FDA.