March 2023 • PharmaTimes Magazine • 9
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Sanofi’s efanesoctocog alfa may be a game-changer for haemophilia A patients
Research has demonstrated positive safety, efficacy and pharmacokinetic credentials of Sanofi’s efanesoctocog alfa drug.
The therapy is an investigational treatment for haemophilia A and the data showed that it delivered a normal to near-normal factor activity levels for the most of the week during a once-a-week dosing pattern.
Haemophilia A is a lifelong, rare and genetic condition affecting the ability of a person’s blood to clot, and occurs in about one in 5,000 male births every year, while being uncommon in females. Individuals can experience bleeding episodes that can cause irreversible joint damage, severe pain and life-threatening haemorrhages.
Data from the vital XTEND-1 phase 3 study demonstrated that efanesoctocog alfa – also known as ALTUVIIIO – met both primary and secondary endpoints, showing clinically meaningful prevention of bleeds and superior bleed protection compared to prior factor VIII prophylaxis treatment.
Dietmar Berger, global head of development and chief medical officer at Sanofi, reflected: “We are steadfast in our commitment to developing novel treatment options that have a meaningful impact for patients.”
He added: “We are hopeful that efanesoctocog alfa will help deliver on this goal by offering unprecedented factor activity levels with once-weekly dosing, fulfilling its potential as a best-in-class therapy for haemophilia A.”
Trio of COVID-19 treatments receive NICE nod
The National Institute for Health and Care Excellence’s (NICE’s) final draft guidance has recommended three COVID-19 treatments as options for adults. They are Pfizer’s Paxlovid, GSK’s Xevudy and Roche’s RoActemra.
The Paxlovid, Xevudy and RoActemra therapies were recommended because there was evidence suggesting they are effective in treating COVID-19. Furthermore, these treatments were found to be cost-effective.
Paxlovid is recommended for adults who do not need supplemental oxygen for COVID-19, Xevudy is suggested for the same group where Paxlovid is contraindicated or unsuitable, while RoActemra is endorsed for treating COVID-19 in adults who are having systemic corticosteroids and need supplemental oxygen or mechanical ventilation.
In addition to the news, NICE has revealed that it is developing a new review process to update its recommendations on the clinical and cost-effectiveness aspects of COVID-19 treatments. This will ensure they are made available more quickly to patients if they demonstrate promise against new variants.
Meanwhile, the organisation will also be launching a public consultation on proposals for the rapid update process from 3 April this year. Monitoring for the process is already in place and NICE has the ability to run an update as a pilot scheme during the consultation period.
HOT & NOT
RMI has provided over 2,000 COVID-19 vaccines throughout Dover. It is part of its ongoing partnership with NHS South, Central and West.
The vaccines are an essential element of the ongoing booster programme, with individuals aged 50 years or over being urged to get the jab. The latest expansion of the service comes after RMI’s campaign in Crawley in 2022, during which the organisation delivered almost 5,000 COVID-19 vaccines in a month.
Jazz Pharmaceuticals has revealed that Epidyolex – a cannabidiol oral solution, developed with GW Pharmaceuticals – has received a vital recommendation for reimbursement by the National Institute for Health and Care Excellence.
The therapy concerns patients of two years of age and above, and relates to the adjunctive therapy of seizures associated with tuberous sclerosis complex.
Gritstone bio – a company focused on developing global vaccinations – has revealed that it has established a clinical trial partnership with the National Cancer Institute.
The collaboration involves an evaluation of an autologous T-cell therapy expressing a T-cell receptor targeting mutated KRAS. It is also in combination with Gritstone’s KRAS-directed vaccine candidate, SLATE-KRAS. Neoantigen vaccine candidate, SLATE-KRAS is an off the shelf therapy and has demonstrated early evidence of efficacy.
Investment company BGF invested £69m across a series of businesses in the UK and Ireland during 2022 – with many operating in life sciences-related companies. This amount included £38m of new investments and £31m of follow-on funding.
In addition to the funding provided by BGF, in 2022 alone the team has helped its portfolio of early-stage companies raise over £500m of funding from third-party investors.
In a blow to patients who currently lack recommended treatment options for chronic heart failure with preserved or mildly reduced ejection fraction, Boehringer Ingelheim and Lilly Alliance’s empagliflozin has failed in to receive a positive recommendation from the National Institute for Health and Care Excellence (NICE).
As yet there are no clinically proven medications recommended by NICE for patients with the condition.
Pfizer has decided to discontinue a significant percentage of participants in the US who had been enrolled in the Vaccine Against Lyme for Outdoor Recreationists (VALOR) phase 3 clinical study.
The research is investigating the safety, efficacy and immunogenicity of Lyme disease vaccine candidate, VLA15. Participants – representing approximately half of the total recruited participants in the trial – are being discontinued following violations of good clinical practice at certain clinical trial sites run by a third-party clinical trial site operator.