April 2021 • PharmaTimes Magazine • 8-9
// MEDICINE //
EC nod for J&J's COVID-19 shot
The European Commission (EC) has granted a conditional marketing authorisation for Johnson & Johnson’s single-dose COVID-19 vaccine in individuals aged 18 years and older, adding a fourth licensed vaccine to the European Union's arsenal to fight the spread of the virus.
The approval was supported by data from J&J’s Phase III ENSEMBLE trial, which demonstrated a 67% reduction in symptomatic COVID-19 disease in participants who received the vaccine compared to placebo.
According to the findings, the vaccine, called COVID Vaccine Janssen, was 85% effective in preventing severe disease across all regions studied, and it showed protection against COVID-19 related hospitalisation and death, beginning 28 days after vaccination.
The European Medicines Agency said the vaccine was found to be 67% effective two weeks after inoculation, with mild side effects. While some rival vaccines seem to offer a higher protection rate, J&J’s offering could not only boost supplies but also simplify vaccination programmes due to its one-dose regimen and ease of transport.
The vaccine is made up of an adenovirus modified to contain the gene for making the SARS-CoV-2 spike protein. The adenovirus passes the SARS-CoV-2 gene into the vaccinated person’s cells, which then use the gene to produce the spike protein. The person’s immune system will recognise the spike protein as foreign and produce antibodies and activate T cells (white blood cells) to target it.
“For more than a year, we have been working around the clock – leveraging the scientific minds, scale and resources of our global organisation to bring forward a COVID-19 vaccine,” said Alex Gorsky, chairman and chief executive at the firm. “We are thrilled with [the] Conditional Marketing Authorisation by the EC, which enables our single-dose vaccine to reach many more communities in need, as we continue to do everything we can to help bring an end to this pandemic.”
J&J said in a statement that it aims to begin delivery of the single-dose vaccine to the EU in the second half of April, and to supply 200 million doses to the EU (plus Norway and Iceland) in 2021.
Sanofi's Pompe disease drug enters EAMS
Sanofi's Pompe disease drug avalglucosidase alfa is the latest medicine to join the UK's Early Access to Medicines Scheme (EAMS), having been issued with a positive scientific opinion from the Medicines and Healthcare products Regulatory Agency (MHRA).
The decision gives patients with the condition who are failing to respond to or are losing response to treatment with Myozyme (alglucosidase alfa) access to an alternative enzyme replacement therapy before it has a full regulatory stamp of approval.
In trials involving patients with late-onset Pompe disease, those treated with avalglucosidase alfa for 48 weeks experienced a greater improvement in respiratory function and also in walking distance (32 metres on average) versus those treated with Myozyme (2m on average), while in infantile-onset disease patients generally showed a stabilisation or improvement in their mobility skills, the MHRA noted.
The drug is currently under review by the European Medicines Agency.
MHRA approves Lilly’s Retsevmo
The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted Eli Lilly’s Retsevmo a conditional marketing authorisation for the treatment of RET fusion-positive advanced lung cancer and thyroid cancer.
The move allows doctors to prescribe Restevmo (selpercatinib) as monotherapy treatment for adults with advanced RET fusion-positive non-small cell lung cancer (NSCLC) who require systemic therapy following prior treatment with immunotherapy and/or platinum-based chemotherapy.
The authorisation also includes the treatment of adults with advanced RET fusion-positive thyroid cancer who require systemic therapy after prior treatment with sorafenib/lenvatinib, and also for adults and adolescents aged 12 years and older with advanced RET-mutant medullary thyroid cancer (MTC) who require systemic therapy following prior treatment.
The decision follows data from the LIBRETTO-00 Phase I/II trial, a single-arm study of over 700 patients with RET-driven cancers. The primary analysis included 105 previously treated patients with NSCLC – 64% of which responded to treatment with an average duration of response of 17.5 months. In previously treated RET-mutant MTC patients, the primary analysis of 55 patients had a 69.1% response rate.
“This is good news for patients living with RET-driven cancers as they will soon have a treatment option that targets RET alterations directly,” said Yvonne Summers, consultant oncologist at The Christie NHS Foundation Trust. "With trial results showing median benefit of 17.5 months, this treatment represents a significant advancement in this growing field,” she added.
RET fusion-positive tumours occur in 1-2% of NSCLC patients, and are more commonly found in people who are below the age of 60 years.
NHS green light for Zolgensma
NHS England and Novartis Gene Therapies have signed a ‘landmark’ deal giving patients with spinal muscular atrophy (SMA) type 1 access to the gene therapy Zolgensma (onasemnogene abeparvovec) on the NHS.
SMA is a rare genetic neuromuscular disease caused by a lack of a functional SMN1 gene. This results in the rapid loss of motor neurones, which in turn affects muscle function – if left untreated, SMA type 1 leads to death or the need for permanent ventilation by the age of two in over 90% of cases.
The deal struck between Novartis and the NHS has secured the therapy at a ‘substantial’ confidential discount – Zolgensma usually has a list price of £1.79 million per dose – with terms of the agreement building on draft recommendations from the National Institute for Health and Care Excellence (NICE) to fast-track access for patients while the final guidance is concluded.
According to NHS England, around 80 patients could potentially benefit from Zolgensma treatment each year, and it is currently working to identify ‘centres of excellence' to provide the full range services required to administer the treatment safely, with new specialist services set to begin treating patients later this year.
“This deal is a life-changer for youngsters with this cruel disease and for their families,” said Simon Stevens, chief executive of NHS England. “Spinal muscular atrophy is the leading genetic cause of death among babies and young children, which is why NHS England has moved mountains to make this treatment available, while successfully negotiating hard behind the scenes to ensure a price that is fair to taxpayers.”