8-9 Medicine News

European regulators have cleared Novartis' Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy (SMA), thus opening the door for access to the only gene therapy cleared for this rare condition.

In Europe each year, around 550-600 infants are born with SMA, a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, which causes rapid and irreversible loss of motor neurons.
Zolgensma is a one-time gene therapy administered designed to address the genetic root cause of the disease by replacing the function of the missing or nonworking SMN1 gene, thereby halting disease progression.
According to AveXis, which developed Zolgensma and was acquired by Novartis in 2018, the therapy has shown “prolonged event-free survival; rapid motor function improvement, often within one month of dosing; and, sustained milestone achievement, including the ability to sit without support, crawl and walk independently – milestones never achieved in untreated Type 1 patients.”
Zolgensma's approval is "a significant milestone for the SMA community, bringing new hope to those impacted by this rare, but devastating disease,” said AveXis president Dave Lennon. “We have met with more than 100 stakeholder organisations across Europe to discuss our 'Day One' access programme to enable rapid access with customisable options designed to work within local pricing and reimbursement frameworks.”
The access programme offers a variety of flexible options that can be 'implemented immediately to support swift access and broad reimbursement' across Europe. It is designed to ensure that the cost of patients treated before national pricing and reimbursement agreements are in place 'align with the value-based prices negotiated following clinical and economic assessments'. The programme offers a variety of customisable options, such as retroactive rebates and deferred payments.
AveXis said it intends to make Zolgensma available across the UK 'as soon as possible', and that evaluation of the therapy by NICE to determine NHS reimbursement is already underway.

The National Institute for Health and Care Excellence is now backing NHS use of Tecentriq (atezolizumab) alongside nab-paclitaxel for treating PD L1-positive, triple-negative, advanced breast cancer, following an 'improved offer' from drugmaker Roche.

In draft guidelines, the Institute said its earlier rejection of the drug was overturned after the company updated its economic model and agreed to provide a larger discount to the list price of the drug following a deal with NHS England and NHS Improvement.
Tecentriq, given with the chemotherapy nab-paclitaxel, is the first immunotherapy that specifically targets triple negative breast cancer where tumours have PD-L1 expression of 1% or more. It is given by injection every two weeks to people who have not had chemotherapy for breast cancer that has spread to other parts of the body and where surgery to remove it is not possible.
According to NICE, the evidence suggests that the combination increases the time before the disease worsens by around 2.5 months compared to placebo plus nab-paclitaxel (7.5 months versus 5.0 months, respectively), and overall survival by around 9.5 months (25.0 months versus 15.5 months respectively).
“This is a major advance in treatment for triple negative breast cancer, the like of which has not been seen in over 20 years,” commented Lesley Hugo, integrated franchise lead – breast cancer at Roche Products Limited.

The European Medicines Agency's human medicines committee (CHMP) has kicked off a ‘rolling review’ of data on the use of Gilead's antiviral remdesivir for the treatment of coronavirus disease (COVID-19).

The move, which will allow the EMA to complete its assessment significantly earlier than with a regular evaluation procedure, rides on the back of preliminary results from the ACTT study, which suggest a beneficial effect of remdesivir in the treatment of hospitalised patients with COVID-19.
Data from the trial released by the US National Institute of Allergy and Infectious Disease indicates that patients given remdesivir had a 31% faster time to recovery than those who received placebo (11 days versus 15 days, respectively.
Results also suggested a survival benefit, with a mortality rate of 8.0% for remdesivir versus 11.6% for the placebo group.
However, the EMA stressed that it has not yet evaluated the full study and thus it is 'too early to draw any conclusions regarding the benefit-risk balance of the medicine'.

The Royal Pharmaceutical Society (RPS) is calling for a change in medicines legislation that would allow community pharmacists to alter prescriptions to help minimise unnecessary treatment delays.

The proposals would enable pharmacists to amend the strength and formulation of the medicine dispensed or supply a generic alternative should that originally prescribed be unavailable, without having to contact the prescriber every time, as is already 'common practice' in Scotland.
“Pharmacists in hospital, and in community pharmacies in Scotland, can already routinely make these alterations to prescriptions for the benefit of patients.
"All pharmacists should be allowed to minimise the impact of medicine shortages on patient care,” said RPS president Sandra Gidley.
“At a time when primary care services are under enormous pressure, it’s right to address this imbalance. We want the UK government to introduce greater flexibility and improve access to medicines by enabling community pharmacists across Great Britain to make these simple changes,” she stressed.

Shionogi’s antibiotic Fetcroja (cefiderocol) is cleared in Europe for the treatment of infections due to aerobic Gram-negative bacteria in adults with limited treatment options. It is the first to provide coverage against all Gram-negative pathogens considered critical priority by the WHO – carbapenem-resistant Acinetobacter baumannii, Pseudomonas aeruginosa and Enterobacteriaceae. It is also the world’s first siderophore cephalosporin that uses the bacteria’s own iron uptake system to gain entry into the cell, acting like a Trojan horse. In Europe 25,000 people die each year from drug resistant bacterial infections.

EU regulators have approved Takeda's Adcetris (brentuximab vedotin) for previously untreated systemic anaplastic large cell lymphoma (sALCL) in combination with CHP (cyclophosphamide, doxorubicin, prednisone). Adcetris is the first targeted therapy approved in first-line sALCL for decades, bringing a new option to those living with this type of peripheral T-cell lymphoma (PTCL). Clearance is based on the Phase III ECHELON-2 study, in which Adcetris plus CHP induced a significant improvement in progression-free survival (PFS) versus standard treatment with CHOP (cyclophosphamide, doxorubicin, vincristine, prednisone).

Novartis' Enerzair Breezhaler, a potential first-in-class triple combination therapy containing indacaterol, glycopyrronium and mometasone, has been backed for approval by the EMA's CHMP for asthma. The decision is supported by efficacy and safety data from over 3,000 patients with asthma in Phase III IRIDIUM study, in which it demonstrated significant improvements in lung function. Enerzair Breezhaler also offers a 'digital companion' consisting of an app and sensor which provides inhalation confirmation, medication reminders and access to objective data, to better support therapeutic decisions and enhanced adherence.

Shares in Newron Pharmaceuticals took a massive hit from the failure of sarizotan to show a clear benefit in patients with Rett syndrome, a severe neurodevelopmental disorder primarily affecting females. Top-line data from the Phase II/III STARS trial of the drug showed that it was not effective on primary or secondary endpoints. The primary endpoint was a percentage reduction in episodes of apnoea during waking time versus placebo, while secondary goals related to respiratory symptoms and motor behaviour. Newron confirmed that on the back of the data it will discontinue sarizotan's development programme.

Blueprint Medicines' Ayvakit (avapritinib) failed to best Bayer's Stivarga (regorafenib) in patients with gastrointestinal stromal tumours (GIST). Top-line results from the Phase III VOYAGER study showed that the drug did not significantly prolong progression-free survival versus Stivarga in patients with third- or fourth-line GIST. US regulators approved Ayvakit in January for adults with unresectable or metastatic GIST harbouring a PDGFR-alpha exon 18 mutation, including PDGFRA D842V mutations, but the firm said it would not pursue further development of the drug in GIST beyond its approved indication.

The CHMP has again rejected D&A Pharma's marketing application for Hopveus (sodium oxybate) for the treatment of alcohol withdrawal syndrome and maintenance of abstinence. The committee felt that the supporting data from three clinical studies did not conclusively demonstrate efficacy, and was concerned over several drawbacks in the design and analysis of the trials, as well as potential misuse and abuse of the medicine. The Agency said its concerns on Hopveus’ effectiveness could not be addressed by further restricting the use of the drug as proposed by the company, and so refusal was therefore confirmed after re-examination.

EU green light for Novartis' Zolgensma

NICE u-turn backs Roche's Tecentriq

Gilead's remdesivir under rolling review

RPS calls for greater prescribing power for pharmacists

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