💉 Sarepta Therapeutics’ Duchenne therapy gets accelerated approval; MPP grants sub-licenses for Nilotinib generics; Diabetes rates only going up
#369 | Filling AMR knowledge gaps; One compound v 12 viruses; Mosquitoes invade Europe
Hello, dear reader. Welcome back to The Kable for a quick look back at the week that was for the life sciences.
Yesterday, the US FDA granted accelerated approval to Sarepta Therapeutics’ Elevidys, which is the first gene therapy for Duchenne muscular dystrophy. The accelerated approval means that a late-stage trial is still ongoing, and if the final results (which come out by the end of the year) confirm success, only then will the therapy receive full approval, with potentially expanded scope of its use. The treatment for the progressive and deadly disease is currently limited to confirmed Duchenne patients of ages 4 through 5 years who can still walk and don’t have any contraindications which might affect the treatment’s effectiveness or safety. This gives Sarepta a market of just about 400 children of that age with the disease in the US. Might explain why the one-time infusion is priced at $3.2 million. One of the most expensive on a single-use basis.
The Medicines Patent Pool (MPP) has signed sub-license agreements with four companies - India-based Eugia, Hetero, and Dr Reddy’s Laboratories, and Indonesia-based BrightGene - to produce generics of Novartis’ Nilotinib (sold under the brand name Tasigna), used to treat chronic myeloid leukaemia. Resulting from a license agreement MPP inked with Novartis in 2022, these are the first sub-license agreements that MPP has signed for cancer treatment. They will permit the manufacturers to produce Nilotinib generics in India and 7 middle-income countries and supply their products in 44 specified territories.
And finally (yep, already), Alphabet spinout SandboxAQ has formally announced its biopharma molecular simulation division AQBioSim. Using AI and quantum technologies, AQBioSim hopes to accelerate R&D to develop treatments for cancers, neurodegenerative diseases, and more. AstraZeneca, Sanofi and UC San Francisco are already working with the division.
The Week That Was
With two months of intense fighting in Sudan, the country has suffered severe loss of life and infrastructure, with affected health facilities not able to deliver essential health care to those who desperately need it. Last week ended with the WHO releasing a new funding appeal for a total of $145 million to address the health needs of those affected by the violence in Sudan.
This week, the saga, which began with cough syrup-linked deaths in the Gambia last year, was back in the spotlight. The WHO revealed that it is working with 6 additional unnamed countries to track potentially lethal children’s medicines. The WHO’s probe into tainted syrups – including cough meds, paracetamol and vitamins – also flagged 20 toxic medicines manufactured by 15 different manufacturers from India and Indonesia.
The Economic Times reported that Naturcold cough syrup – suspected of killing a dozen children in Cameroon – may have been made by India's Riemann Labs. In Sri Lanka, health officials investigated fatalities and complications allegedly linked to India-made medicines, this time, the anaesthetic Bupivacaine.
And the Gambia’s Medicines Control Agency (MCA) appointed Mumbai-based Quntrol Laboratories, an independent inspection and testing company, to issue Clean Reports of Inspection and Analysis (CRIA) for every shipment coming to the country from India.
While some countries have an opioid addiction crisis, others don't have any opioids at all. A new WHO report highlighted unequal access to morphine for medical use and proposed actions which can be taken to improve access.
Wired reported about an insidious pollutant which is making its presence felt in India but isn't being talked about enough. When pollution meets climate change, their affair involves the creation of surface ozone. Exposure to surface ozone can damage airways, aggravate diseases like asthma, and make people more susceptible to respiratory infections. But currently, India isn’t doing enough even to monitor the problem effectively.
This week brought positive news from Bavarian Nordic about its chikungunya virus vaccine; the shot succeeded in phase 3 trials, leading to the production of protective antibodies in close to 90% of all trial participants three weeks after inoculation.
Eli Lilly acquired clinical-stage Dice Therapeutics, which develops oral small-molecule drugs as alternatives to infused or injected biological drugs, for a sum of $2.4 billion.
And finally, the WHO finally caught on to the El Niño action. It forecasted a high likelihood that 2023 and 2024 will be affected by El Niño events, potentially boosting the spread of dengue and other arboviruses like Zika and chikungunya.
Newsworthy
The global diabetes burden. In 2021, 529 million people around the world had diabetes, and if we don’t do anything about it, that number will only go up in every country over the next three decades. A BMGF-funded study, led by researchers at the Institute of Health Metrics and Evaluation at the University of Washington and published in The Lancet, assessed global, regional and national diabetes burdens from 1990 to 2021, projecting disease prevalence up to 2050. The study estimates that by that year, about 1.31 billion people will have diabetes.
From 1990 to 2021, global age-standardised diabetes prevalence went up by 90.5%. By 2050, diabetes rates, particularly those of type 2 diabetes, will increase the world over by 59.7%, but not uniformly. In North Africa and the Middle East, prevalence rates will likely reach 16.8%, and in Latin America and the Caribbean, the rate is expected to touch 11.3%. These numbers are far higher than the projected global rate of 9.8% in 2050. This growth will likely be driven by rising obesity and demographic shifts, as prevalence is higher in older adults. And it may also result in a greater prevalence of heart disease and stroke, both of which are associated with diabetes.
To reverse this trend, we need to address not only risk factors of type 2 diabetes, like obesity but also the social and logistical barriers that restrict people’s access to the medical interventions they need. The Lancet Commission on Diabetes has called for an enhanced reliance on high-quality data, especially in LMICs, so policymakers can better assess risks and outline needs. The WHO has already identified diabetes as one of three target diseases in its Global Action Plan for the Prevention and Control of NCDs; goal 3 of the SDGs also targets a reduction in premature deaths due to diabetes and other NCDs by a third by 2030.
The Lancet study, part of the Global Burden of Disease Study 2021, estimates the diabetes burden stratified by geographical and demographic factors. It highlights the factors contributing to increased nutrition-related NCDs in LMICs, including economic and sociopolitical challenges. It calls for creating change – behavioural and structural – with contributions from policymakers, regulators, educators, public health officials and the medical community.
(The Lancet)
The WHO wants answers. The WHO has published its first Global Research Agenda for Antimicrobial Resistance (AMR) in Human Health, outlining 40 research topics on drug-resistant bacteria, fungi and Mycobacterium tuberculosis which must be answered by 2030, in keeping with the SDGs. The agenda will catalyze research and help frame context-specific strategies to prevent infections and the emergence of resistance. This includes the discovery of new diagnostics, improved treatment regimens, cost-effective data collection methods which can inform policies, and the better application of existing interventions in low-resource settings. With the generated evidence, the WHO hopes to give researchers and funders the information they need to give the world the best possible chance to combat AMR, especially benefiting LMICs.
(WHO)
R&D
One compound to take on 12 viruses. Researchers from the National University of Singapore’s Yong Loo Lin School of Medicine have screened thousands of compounds to discover that the plant-based compound Peruvoside – used to treat heart failure – is also able to treat infections from up to 12 medically important viruses, all of which originate from different virus families. These viruses are responsible for diseases including Covid, the flu, herpes, and hand, foot and mouth disease, but when Peruvoside is modified to act on the GBF1 protein (which is central to viral replication), production of the virus is halted. This broad-spectrum antiviral has very low toxicity and minimal side effects. It could provide a solution to viral diseases which don’t currently have a specific vaccine or therapeutic.
(NUS Medicine)
New disease on the block. A patient from Spain was suffering from severe inflammation of their kidneys, intestines, skin, and other organs. Researchers identified a defect in the patient’s DOCK11 gene involved in cell communication. When three other patients with the same mutations emerged, the researchers saw that the mutations resulted in impaired activation of CDC42 (a protein involved in cell cycle regulation) in all 4 patients. They were able to show that the DOCK11 protein is needed for B cells to develop properly, and they linked its absence to T lymphocyte overactivation, potentially leading to tissue and organ damage. All of these findings have led to the discovery of a rare new disease. Three of the four patients mentioned here passed away during the evaluation, but the scientists hope that their discovery will lead other scientists to build on their work and eventually develop a treatment for the condition. Currently, they suspect haematopoietic stem-cell transplants or gene therapies may help.
(The New England Journal of Medicine)
The Kibble
Predicting hits. Freud would have loved seeing scientific proof of how much more insightful your subconscious state is compared to your conscious mind. Researchers have found that individuals’ self-reported enjoyment of a song is not necessarily a good indicator of its popular success. However, when the researchers used wearable devices to collect information about people’s neurophysiology while they listened to a song, and trained an ML algorithm to analyze those responses, they were able to much better predict how popular a song might be. It only took 33 participants to guess which songs millions of others might like. Musicians, are you listening?
(Frontiers in Artificial Intelligence)
Bottom line
Come climate change, come viral diseases. European CDC officials are warning about a growing risk of mosquito-borne viral diseases in Europe, and it’s all thanks to climate change. As Europe experiences a warming trend, the European CDC has said that conditions are becoming more favourable for invasive mosquito species like Aedes albopictus and Aedes aegypti to make comfy European homes for themselves. Longer summers, more heat waves, and frequent flooding could lead to an increased prevalence of dengue, chikungunya, Zika, and other diseases.
While a decade ago, the Aedes albopictus mosquito had developed a self-sustaining population in 8 European countries, affecting 114 regions, now, the mosquito is established in 13 countries and 337 regions. So as Europe gets a taste of what warmer countries have been going through all this while, the ECDC is calling for the elimination of standing water, the use of eco-friendly larvicides, and a number of other interventions to control mosquito population growth and promote personal protection.
(ECDC)
Oh, and Gopal Nair doesn't want you to see this.