The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has become the first regulatory agency to approve Lagevrio (molnupiravir), developed by Ridgeback Biotherapeutics and Merck & Co – known as MSD outside the US and Canada.

The antiviral drug is the first oral antiviral drug approved to treat COVID-19. The MHRA has approved its use for those with mild to moderate disease and at least one risk factor for developing severe illness. These risk factors include obesity, older age (>60 years), diabetes mellitus or heart disease.

“This will be a game changer for the most vulnerable and the immunosuppressed, who will soon be able to receive the groundbreaking treatment,” said the UK health and social care secretary, Sajid Javid. “The UK is leading the way to research, develop and roll out the most exciting, cutting-edge treatments.

June Raine, chief executive at the MHRA, said: “It is the world’s first approved antiviral for this disease that can be taken by mouth rather than administered intravenously. This is important, because it means it can be administered outside a hospital setting, before COVID-19 has progressed to a severe stage.”

Merck is already producing Lagevrio and expects to produce 10 million courses by the end of the year and at least 20 million courses in 2022.

The approval is based on results from the phase 3 MOVe-OUT clinical trial, which showed that molnupiravir reduced the risk of hospitalisation or death by approximately 50% against placebo.

Both the US Food and Drug Administration and European Medicines Agency, along with other regulatory bodies around the world, are reviewing the data on the drug.

The approval by the US Food and Drug Administration (FDA) of Boehringer Ingelheim’s Cyltezo as an interchangeable biosimilar for Humira in certain inflammatory diseases looks set to make an already crowded field even more competitive.

The key difference with this approval is that an interchangeable biosimilar product may be substituted for the reference product without the prescriber having to change the prescription. This can happen at the pharmacy in US states that permit pharmacy-level substitution.

The FDA approval covers adult patients with forms of rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn’s disease, ulcerative colitis and plaque psoriasis.
The Humira market is worth more than $15bn annually and, in addition to Boehringer’s Cyltezo, other biosimilars that are set to launch in 2023 include Amgen’s Amjevita, Sandoz’s Hyrimoz, Pfizer’s Abrilada and Samsung’s Hadlima.

The 2023 timeline is the result of intense legal wrangling with Humira’s maker AbbVie that ended with a 2019 agreement to delay the launches of adalimumab biosimilars. One potential disruptor to this carefully laid plan is Alvotech, which has not settled its dispute with AbbVie and has also completed a successful switching study.

In Europe, four adalimumab biosimilars have launched since 2018, with nearly 35% of European patients switched from Humira to a biosimilar by the end of 2019, despite AbbVie reducing its price for the drug.

To date, the FDA has approved 31 biosimilar products, including two interchangeable products, for a variety of health conditions.

High-level results from the phase 3 TOPAZ-1 trial showed that AstraZeneca (AZ)’s PD-L1-blocking monoclonal antibody Imfinzi (durvalumab) plus chemotherapy demonstrated a clinically meaningful survival benefit over chemotherapy alone as a first-line treatment for advanced biliary tract cancer (BTC).

More than 200,000 people each year are diagnosed with BTC, a group of rare and aggressive cancers in the bile ducts and gall bladder. The prognosis is generally poor, with only 5-15% of patients surviving five years.

The trial was unblinded early due to “clear evidence of efficacy”, said Susan Galbraith head of oncology R&D at AstraZeneca. “We have now delivered two positive gastrointestinal cancer trials in a row for Imfinzi, following the HIMALAYA trial in liver cancer.

“We believe the significant survival benefit demonstrated marks a new era of immunotherapy treatment in this devastating disease,” she added.

TOPAZ-1 involved 685 patients with unresectable advanced or metastatic BTC in more than 145 centres across 17 countries. The high-level results also show an improvement in progression-free survival and overall response rate, both key secondary endpoints in the trial.

Earlier this month, AZ released new data from the ongoing HIMALAYA phase 3 trial studying the most common form of liver cancer, hepatocellular carcinoma (HCC). The trial – which involved a ‘priming’ dose of AZ’s experimental human monoclonal antibody, tremelimumab – demonstrated a statistically significant and clinically meaningful overall survival benefit versus sorafenib.

While there are more than 7,000 rare diseases, only two gene therapies have been approved by the US Food and Drug Administration (FDA) to treat inheritable conditions.

Bringing together ten pharma companies with the FDA, National Institutes of Health and five non-profit organisations, the Bespoke Gene Therapy Consortium (BGTC) aims to ‘optimise and streamline’ the gene therapy development process. The hope is this initiative will accelerate the development of treatments for the 30 million Americans suffering from a rare disease.

The new body recognises that the development of gene therapies for rare diseases is highly complex, time-consuming and expensive, and that the development process is ‘stymied by limited access to tools and technologies, lack of standards across the field, and a one-disease-at-a-time approach to therapeutic development’.

It aims to develop a standardised therapeutic development model based around a common gene delivery technology (or vector) to save time and money in developing specific gene therapies.

Five major pharma companies have signed up to be part of the BGTC – Biogen, Janssen, Novartis, Pfizer and Takeda – plus smaller companies like REGENXBIO, Spark Therapeutics, Taysha Gene Therapies and Ultragenyx, as well as Thermo Fisher Scientific. The non-profit partners include the Alliance for Regenerative Medicine, the American Society of Gene and Cell Therapy and the National Organization for Rare Disorders.

Merck & Co – known as MSD outside the US and Canada – has received its first European approval for Keytruda in a breast cancer setting based on survival data from the phase 3 KEYNOTE-355 trial.

Keytruda can now be used as first-line treatment in combination with chemotherapy to treat locally recurrent unresectable or metastatic triple-negative breast cancer in adults whose tumours express PD-L1 and who have not received prior chemotherapy for metastatic disease.

Triple-negative breast cancer is an aggressive form of the disease with a high recurrence rate and more commonly diagnosed in in people who are younger than 40, who are black or who have a BRCA1 mutation.

In the KEYNOTE-355 trial, Keytruda plus chemotherapy saw a 27% reduction in the risk of death and a 34% reduction in the risk of disease progression or death compared to chemotherapy alone.

Patients in all 27 European Union member states plus Iceland, Lichtenstein, Norway and Northern Ireland can now receive Keytruda in combination with chemotherapy.
The company has the largest immuno-oncology clinical research programme with more than 1,600 trials studying Keytruda across a wide variety of cancers and treatment settings currently underway.

Keytruda is approved in the same indication in the US as well as for patients with high-risk, early-stage TNBC in combination with chemotherapy as neoadjuvant treatment.