November 2020 • PharmaTimes Magazine • 8-9
// MEDICINE //
NHS patients gain access to Novartis' MS drug
Cost regulators for NHS treatments in England and Wales and Scotland have backed funding for use of Novartis' Mayzent (siponimod) in patients with secondary progressive multiple sclerosis (SPMS).
The decision allows routine prescribing of the first oral disease-modifying therapy to delay disability progression and cognitive decline in patients with active disease.
Around 38,000 people in the UK are affected by SPMS, which constitutes over a third of all people living with MS. Often, diagnosis is delayed or avoided because of uncertainty surrounding the progression of relapsing/remitting multiple sclerosis (RRMS) to SPMS, which leaves many patients without the most effective treatment.
Mayzent is a selective small-molecule agonist of sphingosine-1-phosphate (S1P) receptors S1P1 and S1P5 which, in the Phase III EXPAND study, was shown to reduce the risk of three-month confirmed disability progression by a statistically significant 21% versus placebo.
In the SPMS active disease subgroup, results demonstrated that Mayzent cut the risk of both three- and six-month CPD by 31% versus placebo, respectively.
“The decision by both NICE and the SMC represents a significant step forward in ensuring people living with SPMS with active disease in the UK have access to treatment,” said Chinmay Bhatt, managing director UK, Ireland & Nordic for Novartis Pharmaceuticals. “We are working closely with the NHS to ensure eligible patients can start benefiting from Mayzent as soon as possible.
PIM status for Sanofi's Pompe disease treatment
The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has issued Sanofi a Promising Innovative Medicine (PIM) designation for avalglucosidase alfa in the treatment of Pompe disease.
A PIM designation is awarded to treatments that could offer a major advantage for patients and is an early indication that a medicine is a promising candidate for the Early Access to Medicines Scheme (EAMS), which enables patients to access certain therapies before they are officially approved.
The decision was partly based on data from the Phase III COMET trial, which evaluated the safety and efficacy of avalglucosidase alfa compared to alglucosidase alfa – the standard of care – in patients with late-onset Pompe disease. The trial met its primary endpoint, showing non-inferiority of avalglucosidase alfa in improving respiratory muscle strength compared to the standard of care.
The PIM designation “not only recognises the life-threatening nature and high unmet need of Pompe disease but indicates that avalglucosidase alfa is an innovative medicine which could offer a potential new therapeutic option for patients with this rare disease,” said Nicole Farmer, general manager UK & Ireland, Sanofi Genzyme.
Pompe disease belongs to a group of rare and genetically inherited diseases called lysosomal storage disorders, which results in the deficiency of the acid alpha-glucosidase enzyme. This causes the accumulation of glycogen in certain organs and tissues, and impairs their ability to function normally.
Pharmacists call for counterfeit meds action
The Royal Pharmaceutical Society of Great Britain (RPS) has called on the UK government to take immediate action to fight counterfeit medicines from entering the country following the UK’s exit from the European Union.
In a letter addressed to Health Secretary Matt Hancock, the RPS emphasised the need for rigorous plans to be put in place to maintain formal links with the EU, to help authenticate the legitimacy of medicines that move between the two territories.
Under current plans, the UK will cease to benefit from the provisions of the Falsified Medicines Directive (FMD), which ensures that medicines in the EU are legitimate, safe and high-quality. The RPS has presented concerns that this could leave the UK vulnerable to a swarm of counterfeit medicine entering the supply chain, which could impact patient care in the UK and across the EU.
“In our letter to the government we have emphasised that establishing technical agreements with the EU is now more critical than ever in our fight against counterfeit medicines. We have made it clear that the ideal way forward is for continuity of the provisions of the Falsified Medicines Directive, enabling ongoing connectivity between the UK and Europe,” said Sandra Gidley, president of the RPS.
“Not only will this help to provide assurances to patients about the safety of their medicines but it will also ensure the UK can continue to benefit from the significant investment made by the NHS, pharmacy organisations and individual pharmacy owners in the infrastructure for FMD,” she added
Paediatric neuroblastoma treatment to enter clinical trials
A new treatment for neuroblastoma will be advanced into paediatric clinical trials by the Institute of Cancer Research, London, by the end of 2020, raising hopes of a new approach to fighting the aggressive childhood cancer.
Fadraciclib, which has already passed safety trials in adults, indirectly targets N-Myc, a gene that occurs in aggressive forms of neuroblastoma. It works by blocking N-Myc activity by switching off the production of the gene.
In mice, the drug slowed down and stabilised tumour growth, and also extended survival. When combined with chemotherapy, it shrank mice tumours to the point of virtually eradicating them.
“We welcome the news that a potential new treatment will be advanced to clinical trial and reach more young cancer patients,” said Tony Heddon, chair of research charity Neuroblastoma UK. “Less than 50% of children with high-risk neuroblastoma will survive for five years or more after their diagnosis. And for those children who do survive, the drugs used to save them may cause long-lasting damage.
“There is a real need to provide less toxic and more effective treatment for our children. This important development offers real hope to families affected by this life-threatening cancer,” he added.