Biotechnology companies Biogen and Amicus have won the two top prizes for their rare disease medicines at the UK Prix Galien 2018 award ceremony held at London’s House of Commons.

Biogen’s Spinraza (nusinersen) has won the Orphan Product award while Galafold (migalastat), developed by Amicus, received the Innovative Product medal for its ability to treat patients with rare diseases.

Except for Amicus’ Galafold, all other medicinal products shortlisted for final were for orphan conditions.

Galafold became the first orphan product in the history of the UK Prix Galien to receive the innovative product award.

UK Prix Galien Judges chair Sir Michael Rawlins said: “The quality and depth of innovation that emerges from the UK life sciences industry continues to change lives and make a real difference to people all over the world.

“That innovation is the reason why UK Prix Galien exists – to honour excellence in medical research and development (R&D) and celebrate the achievements of all those on whom we rely for the discovery and development of new innovation.”

Biogen’s Spinraza is a globally licenced medicine developed for the treatment of spinal muscular atrophy (SMA), a serious neurodegenerative disease that results in debilitating muscle weakness.

Nusinersen has already won five Prix Galien awards in the US, Italy, the Netherlands, Belgium, Luxembourg and Germany.

Amicus’ Galafold is an oral medication that can be used for the treatment of patients with Fabry disease, which is a rare lysosomal metabolic storage condition that causes progressive, irreversible nervous-system disease, cardiac problems, renal disease and stroke.

In addition, US-based medical device company Medtronic’s Solitaire was awarded a commendation in the newly introduced Medical Technology category.

Health think tanks have warned that the UK National Health Service (NHS) is at risk of facing shortage of approximately 350,000 staff by 2030.

A new briefing by the King’s Fund, Nuffield Trust and Health Foundation said that the shortages could affect care quality and the budget for frontline services.

The warning follows a recent analysis by Nuffield Trust, which said that the NHS may experience up to £2.3bn in extra annual costs by the end of 2019-2020, in case of a no-deal Brexit.

According to the latest briefing titled ‘The health care workforce in England: make or break?’, the health service currently has a staff shortfall of more than 100,000. The think tanks predict that this may increase by nearly 250,000 by 2030.

The think tanks said: “If the emerging trend of staff leaving the workforce early continues and the pipeline of newly trained staff and international recruits does not rise sufficiently, this number could be more than 350,000 by 2030.”

The current shortages are believed to be due to incoherent approach to workforce policy at a national level, inadequate funding for training and poor workforce planning, among others.

A previous report published in Research Professional revealed that the NHS staff shortages could lead to cutbacks in clinical trials, which in turn may impact drug research activity.

As the blueprint for an additional £20.5bn funding for the NHS over the next ten years is set to be published in the near future, the think tanks highlight the need for creating a long-term plan to address the workforce crisis.

The briefing noted: “Unless new staff can quickly be recruited and trained, the NHS simply will not have the workers available to meet the demand for healthcare expected over the next decade, exacerbating recruitment and retention problems.

“This will mean that even with the extra money to commission frontline services, healthcare providers will not have the staff to deliver them.”

A clinical trial led by Walter and Eliza Hall Institute of Medical Research in Australia has demonstrated that genomic profiling could help match rare cancer patients with appropriate treatments.

Conducted at four sites across Australia, the patient-driven NOMINATOR trial assessed if genomic sequencing of rare cancers can enable better diagnosis and treatment.

The trial investigators sequenced a panel of genomic markers in cancers to identify molecular features or mutations that can be targeted with treatments currently available for other cancer types with similar characteristics.

The pilot study included 36 patients. The study showed that genomic profiling yielded meaningful information in more than 50% of the participants, which influenced diagnosis and treatment.

NOMINATOR has been designed as a two-year study and is set to enrol 100 rare cancer patients with few standard treatment options.

Walter and Eliza Hall Institute researcher Clare Scott said: “Australians in this trial came to us after they had exhausted all their options. Using genomic profiling we were able to uncover new information that gave many patients new treatment options – and ultimately, new hope.

“Genomic profiling provided meaningful information that influenced diagnosis and treatment in around half of the participants. 20% of those tested got a new treatment plan as a result and 6% of participants were given a new diagnosis.”

A cancer type is considered to be rare if fewer than six out of 100,000 people are affected per year.

In Australia, rare cancers constitute more than 20% of cancer diagnoses and are said to be collectively responsible for more cancer deaths than any single cancer type.

The researchers believe that this type of cancer requires better treatment options, and these early trial results could be a step towards meeting these needs.

Clinical Oncology Society of Australia president Phyllis Butow said: “Around 52,000 Australians are diagnosed with rare or less common cancers each year. Those directly affected by the disease helped call for and fund this research, so it’s great to see these initial promising results being presented to cancer experts from across the country.”

A survey by the World Health Organization (WHO) has revealed huge variations in the use of antibiotics across the world, with certain countries recording very high levels of consumption.

The WHO Report on Surveillance of Antibiotic Consumption, published during World Antibiotic Awareness Week, gathered data on antibiotics use in 2015 across 65 countries and areas.

WHO said that the data could provide better insights into the patterns and volume of antibiotics use at national level, in turn supporting policies and regulations to optimise consumption.

A measure called defined daily dose (DDD), the average dose taken by a patient per day, was used to compare the medicine consumption between countries.

Six African countries, six in the Americas and 46 European countries participated in the survey. Overall consumption of antibiotics in these countries and areas varied from 4.4 to 64.4 DDD per 1,000 inhabitants per day.

Burundia reported the lowest antibiotics use with 4.44 DDD per 1,000 inhabitants. Meanwhile, Mongolia had the highest of 64.41 DDD per 1,000 inhabitants, and was followed by Iran which recorded 38.78.

In the European region, Azerbaijan had the lowest rate of 7.66 DDD per 1,000 inhabitants. Turkey stood at the top with 38.18 DDD per 1,000 inhabitants.

The UK had a higher than average consumption at around 20 DDD per 1,000 inhabitants per day.

In the report, WHO said: “The use of antibiotics appears to be very high in some parts of the world, suggesting their overuse, whereas it is low in others, which may indicate limited access to these life-saving medicines.

“Findings from this report confirm the need to take action to ensure that antibiotics are used appropriately, such as enforcing prescription-only policies and implementing antimicrobial stewardship programmes.”

The report further looked at the type of antibiotics used. Access category of antibiotics, including amoxicillin and amoxicillin/clavulanic acid, were found to occupy more than 50% of the total amount consumed.

However, the proportion of Broad spectrum antibiotics, belonging to Watch category, varied around 20%-50% across countries.

The report showed that reserve group antibiotics, which are to be used as ‘last resort’ for specific indications, contributed less than 2% of the total antibiotics used in the majority of the high-income countries.

Certain countries, including the US, China and Southeast Asian regions, such as India, were not included in the survey.

The organisation added: “WHO aims to increase the number of countries participating in the global programme on surveillance of antimicrobial consumption and to continue supporting low and middle-income countries in their efforts to build and improve surveillance systems on antimicrobial consumption adapted to the national context.”

US-based biotechnology firms BioTime and Asterias Biotherapeutics have entered into a definitive merger agreement to form an integrated company focused on cell therapies.

Under the terms of the agreement, BioTime will buy all outstanding Asterias shares for an undisclosed sum.

As per the agreement, BioTime will offer 0.71 of its shares for each share of Asterias, which will hold approximately 16.2% interest in the combined entity.

BioTime CEO Brian Culley said: “Our vision is to build BioTime into a premier cell therapy company and this acquisition can support that transformation as it not only diversifies our pipeline with two additional clinical-stage assets addressing high unmet medical needs, but also adds partnerships with notable institutions such as the California Institute for Regenerative Medicine and Cancer Research UK.”

Culley added that the merger will enable the company to address disease areas that require novel therapeutics.

Asterias Biotherapeutics pipeline consists of an oligodendrocyte progenitor cells (OPCs)-based cell therapy, called OPC1, being developed for the treatment of severe spinal cord injury. OPC1 is currently undergoing a Phase I/IIa clinical trial.

In addition, the company is developing the VAC2 cancer immunotherapy candidate in non-small cell lung cancer (NSCLC). It is being evaluated in a Phase I trial funded and conducted by Cancer Research UK.

Asterias Biotherapeutics CEO Michael Mulroy said: “The stock merger structure provides Asterias stockholders the ability to continue their investment in our clinical programmes in spinal cord injury and non-small cell lung cancer as part of a larger, more diversified company with greater resources.”

The merger, subject to customary closing conditions and approvals, is expected to close in the first quarter of next year.

Novartis unit Sandoz has voluntarily recalled one lot of Losartan Potassium-Hydrochlorothiazide tablets after finding traces of impurity in the API Losartan manufactured by Zhejiang Huahai Pharmaceutical (ZHP).

The N-nitrosodiethylamine (NDEA) impurity is classified as a potential human carcinogen by the International Agency for Research on Cancer (IARC).

Losartan Potassium-Hydrochlorothiazide tablets are indicated for the treatment of hypertension as a monotherapy or in combination with other antihypertensive agents.

Sandoz said that the recalled lot of its products, which are produced by Lek Pharmaceuticals in Slovenia, has so far not led to any adverse event reports.

The company is notifying distributors and retailers and asking them to immediately cease distribution of the identified batch.

It also advised patients to seek alternative therapy before returning their current medication.

Sandoz in a statement said: “Patients who are on Losartan Potassium-Hydrochlorothiazide should continue taking their medication, as the risk of harm to a patient’s health may be higher if the treatment is stopped immediately without any alternative treatment.

“Patients should contact their physician or healthcare provider if they have experienced any problems that may be related to taking or using Losartan Potassium-Hydrochlorothiazide.”

NDEA was previously discovered by the European Medicines Agency and the US Food and Drug Administration (FDA) in September in multiple batches of drugs containing valsartan as the active pharmaceutical ingredient (API).

ZHP reported the presence of this impurity in several batches of its valsartan API. The FDA also found it in three lots of Torrent Pharmaceuticals’ recalled valsartan products.

Spain’s healthcare regulator the Agency of Medicines and Medical Devices (AEMPS) has published a review of popular pain drug Nolotil (metamizole) following rising complications and deaths associated with the side effects of the medications in British patients.

The regulator’s inquiry states that Nolotil should not prescribed for people who are only in the country for a short time, particularly tourists from the UK.

One of the major side effects of Nolotil is agranulocytosis. This causes the brain marrow to not produce enough white blood cells, and can result in fatal blood poisoning. AEMPS particularly identified two groups most at risk from this condition: the elderly and those with suppressed immune systems.

According to Spanish newspaper El Pais, AEMPS’ note said: “The AEMPS has reviewed the situation in Spain due to recent reports of agranulocytosis filed with the Spanish Pharmaceutical Monitoring System, particularly in patients of British origin.

“While there has been a years-long debate around a greater sensitivity among the population of northern Europe, and certain genetic factors have been studied, available information does not allow us to either rule out or confirm a greater risk for groups with specific ethnic characteristics.”

AEMPS continued to say the drug should only be taken for “short-term treatments, seven days at the most” and in cases where it needs to be taken for longer, patients must be monitored, and if this is not possible, such as for tourists, then it should not be prescribed.

This review follows a campaign organised by the families affected and a medical translator based in Valencia called Cristina Garcia del Campo. Campo submitted evidence to AEMPS of 120 cases of mainly British patients she had translated for who had experienced side effects when taking Nolotil.

There have been previous reports, including that by the Internal Medicine Unit of the Costa del Sol Hospital, which observed that agranulocytosis due to Nolotil was more common in British patients than in Spanish ones.

The authorities have not determined why northern European patients seem to react more severely to the drug than Spanish and Latin American patients.

In a statement to The Times, the manufacturer of Nolotil Boehringer Ingelheim said that there was no scientific evidence that certain populations were more prone to develop side effects of the drug.

The company also told El Pais: “We are working closely with the AEMPS to provide information about the effectiveness and safety of this drug.”

Despite being a very popular pain medication in Spain and Latin America, Nolotil is not licensed for use in the UK, the Republic of Ireland, the US or Sweden, among other countries.

Australian biotechnology firm Sementis has entered into a research and development (R&D) partnership with UK-based Enesi Pharma for the development of potential vaccine candidates to treat peanut allergy and chikungunya/Zika infection.

Under the alliance, the partners will leverage Enesi Pharma’s ImplaVax needle-free implant and device technology to create and assess solid dose versions of Sementis’ peanut hypoallergy vaccine and single vectored chikungunya/Zika vaccine candidates.

Designed for subcutaneous vaccination, ImplaVax technology is said to enable better immune responses than conventional injection protocols.

Enesi Pharma CEO David Hipkiss said: “The collaboration to develop a solid dose version of these vaccines for use with our ImplaVax needle-free device offers the potential to deliver a robust and effective product addressing significant global health challenges.

“We are seeing increasing interest from all areas of the vaccine and public health industries based on the potential benefits that ImplaVax could provide to improve vaccination.”

The investigational peanut hypoallergy vaccine is expected to provide a permanent cure for the allergy. Its ImplaVax-enabled variant is meant to facilitate a simple and effective option on a large scale.

Sementis is developing the single vectored chikungunya/Zika vaccine to protect from Zika and chikungunya virus infections with a single vaccination shot.

An ImplaVax-SCV chikungunya/Zika vaccine is said to provide the potential for long-lasting immunity, while retaining potency across multiple storage conditions for prolonged duration.

Sementis CEO Dr Paul Howley said: “We are excited with this collaboration as we are very confident that the ImplaVax technology will work extremely well with our vaccine vector technology to reduce, or even eliminate, the reliance on maintaining a cold-chain environment from manufacture to point-of-care, and possibly providing long-term stability to our vaccines.”

The terms of the collaboration will see Enesi Pharma devise solid dose implants based on SCV vaccine provided by Sementis.

Meanwhile, Sementis will be responsible for safety and immunogenicity testing, as well as preclinical development.

Enesi Pharma signed a similar R&D alliance with the UK’s Public Health England (PHE) agency last month over needle-free solid-dose vaccine products for emergent threat pathogens.