June 2021 • PharmaTimes Magazine • 8-9
// MEDICINE //
NICE green light for Ultomiris
NHS patients with paroxysmal nocturnal haemoglobinuria (PNH) or atypical haemolytic uraemic syndrome (aHUS) are to get access to treatment with Alexion's Ultomiris (ravulizumab) following a green light from cost regulators.
The National Institute for Health and Care Excellence (NICE) has published final guidance recommending NHS use of the drug for PNH and draft guidance backing its use to treat aHUS.
PNH is characterised by an excessive breakdown in red blood cells, which can lead to anaemia, kidney problems and blood clots in the blood vessels. aHUS causes blood clots in small blood vessels, which can lead to organ damage and significant kidney impairment, thrombosis, heart failure and brain injury.
To date, people with symptomatic PNH or aHUS have only have one treatment option available on the NHS – Alexion's Soliris (eculizumab) – which is delivered intravenously every two weeks. On the back of NICE's approval, patients with either disease will now be eligible for treatment with Ultomiris, which is administered every eight weeks and thus could improve the quality of life for people with these rare blood disorders.
According to NICE, clinical trial evidence shows that, in people with PNH, treatment with Ultomiris is “at least as clinically effective as eculizumab”, and causes “fewer episodes of breakthrough haemolysis, a symptom of PNH, reducing the number of hospital admissions and the need for blood transfusions”. Also, indirect comparisons of Ultomiris with Soliris for people with aHUS similarly suggest that Ultomiris is as effective at treating the condition.
“We are hopeful that the increased time between doses with this new treatment will lead to a better quality of life for these individuals and their loved ones,” said Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE.
Around 270 people with PNH and 160 people with aHUS will be eligible for the new treatment.
New gene therapy leads CHMP approvals
The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has backed approval of bluebird bio’s gene therapy Skysona (elivaldogene autotemcel) for the treatment of early cerebral adrenoleukodystrophy (CALD).
If approved, Skysona will be the first one-time gene therapy approved to treat patients with CALD who do not have a matched sibling haematopoietic stem cell donor.
A further seven medicines were put forward by the CHMP: Albireo’s Bylvay (odevixibat) for the treatment of progressive familial intrahepatic cholestasis (PFIC); Rhythm Pharmaceuticals’ Imcivree (setmelanotide) for the treatment of obesity and the control of hunger; Almirall’s Klisyri (tirbanibulin mesylate) for non-hyperkeratotic, non-hypertrophic actinic keratosis; Bioprojet Pharma’s Ozawade (pitolisant) for excessive daytime sleepiness in obstructive sleep apnoea; Gedeon Richter’s Ryeqo (relugolix/estradiol/norethisterone acetate) for the symptoms of uterine fibroids; Bayer’s Verquvo’s (vericiguat) for symptomatic chronic heart failure in adults with reduced ejection fraction; and Icatibant Accord (icatibant) for the treatment of acute attacks of hereditary angioedema.
Nod for Tagrisso under Project Orbis
AstraZeneca's post-surgery therapy for lung cancer, Tagrisso (osimertinib), is the first drug to receive approval from the UK's Medicines and Healthcare products Regulatory Agency (MHRA) under Project Orbis.
Project Orbis is a programme coordinated by the US Food and Drug Administration (FDA) with Canada, Australia, Switzerland, Singapore, Brazil and the UK as other participants, which offers a framework for concurrent submissions and collaboration reviews for promising cancer treatments.
Tagrisso is licensed treatment for patients with mid and later stage non-small cell lung cancer (NSCLC) who test positive for a specific gene mutation called EGFR, which occur in around 12% of lung cancer patients.
The treatment scope of the drug has now been expanded under Project Orbis to include a new population of patients in early-stage disease, offering a novel treatment option for these patients after their cancer has been surgically removed.
NHS England, NICE and AstraZeneca have reached an agreement to enable early access to the drug for early-stage lung cancer patients in England "on a budget-neutral basis to the NHS while NICE undertakes its appraisal", the MHRA noted.
“With Project Orbis, we are working to ensure that patients receive earlier access to promising, life-saving cancer treatments,” said Dr June Raine CBE, chief executive of the MHRA. “We know that the earlier we can treat patients, the better their outcomes.”
“Project Orbis is a powerful example of how collaboration between regulatory authorities around the world can accelerate the approval of life-changing treatments and we’re delighted that osimertinib is the first medicine to undergo this innovative review process with the MHRA,” added Tom Keith-Roach, president of AstraZeneca UK. “It’s fantastic news that NHS patients in England with this specific type of early-stage lung cancer will have early access to this medicine, which could significantly improve their chance of disease-free survival.”
AI-designed Alzheimer's drug enters Phase I
An Alzheimer’s disease drug candidate designed using Exscientia’s artificial intelligence (AI) technology has now entered Phase I clinical testing.
The drug – DSP-0038 – is being advanced by Japanese pharma company Sumitomo Dainippon Pharma as part of a collaboration with Oxford, UK-based Exscientia, with initial evaluation for the treatment of Alzheimer’s disease psychosis.
DSP-0038 is the third molecule created using Exscientia’s AI technologies to enter the clinic, following DSP-1181, which is being developed with Sumitomo Dainippon to treat obsessive compulsive disorder, and the immuno-oncology agent EXS-21546.
Exscientia and Sumitomo Dainippon designed DSP-0038 as a single small molecule that exhibits high potency as an antagonist for the 5-HT2A receptor and the 5-HT1A receptor, while selectively avoiding similar receptors and unwanted targets.
The drug candidate will be assessed for improved antipsychotic effects associated with Alzheimer’s psychosis, in addition to improvements in behavioural and psychological symptoms of dementia including agitation, aggression, anxiety and depression.