💊 US FDA approves haemophilia A gene therapy; Novartis sells its dry eye drug; Australia approves medical trips

#375 | EU prepares for vaccine apartheid; Maiden Pharma gets sued; CRISPR has competition

Hello, dear reader. Welcome back to The Kable, where we're kicking off the month with quite a packed edition.

Last week, the US FDA approved BioMarin Pharmaceutical’s Roctavian, a gene therapy for severe haemophilia A. It was approved in the EU in August last year. There are already other effective treatments on the market in the US, but this one-time therapy, priced at $2.9 million, will allow patients to avoid regular treatments over their lifetime. Also, in a first, the FDA has approved BioMarin and ARUP Laboratories’ AAV5 DetecCDx test as a companion diagnostic to identify which patients would be best served by the treatment.

In the UK, Eli Lilly has received marketing authorization for its ulcerative colitis treatmentMirikizumab.

Meanwhile, a small biotech company CellTrans has received the first FDA approval for a type 1 diabetes cell therapy. The therapy uses pancreatic islet cells sourced from deceased donors.

A group of researchers led by Novartis scientists has identified a new class of antiparasitics called cyanotriazoles. These compounds could be used to develop cheap oral pills to treat sleeping sickness, Chagas disease and leishmaniasis.

Ipsen and Genfit are reporting success in their phase 3 trial of Elafibranor for the treatment of the rare liver disease, primary biliary cholangitis.

Bausch + Lomb is paying Novartis $1.75 billion upfront, plus potential milestone payments of up to $750 million, for the latter's dry eye disease drug Xiidra and two experimental ophthalmology products.

China’s CSPC Pharma Group is partnering with Pfizer to launch a local brand of oral Covid treatment.

Sandoz has launched a biosimilar version of AbbVie’s arthritis treatment Humira. Meanwhile, Organon and Samsung Bioepis have also launched an 85% cheaper copycat version of the drug.

Germany’s BioMed X is beginning its first research project with Sanofi to develop a computational platform to accurately predict the efficacy of first- or best-in-class drug candidates in virtual patient populations and accelerate drug development.

An FDA inspection of India-based Intas Pharmaceuticals’ manufacturing facility has uncovered instances of document manipulation and cleaning violations. This facility produces the cancer drug Cisplatin, which is currently in short supply.

Medtronic has found a vulnerability in its heart device data management system which could allow hackers to access patient data.

The American Society of Anaesthesiologists says that people taking popular diabetes and weight loss drugs like Ozempic should temporarily halt taking them before elective surgery. This warning comes amidst anecdotal reports that patients taking the drugs may be at increased risk of vomiting and aspirating food into the lungs during general anaesthesia.

Australia has become the first country to allow doctors to prescribe psilocybin and MDMA to treat psychiatric conditions. Nonetheless, there are concerns regarding the inconclusiveness of relevant research and insufficient regulation.

The WHO has released updated guidance on the prevention and management on malnutrition in children. This update, for the first time, includes instructions on severe and moderate forms.

The World Bank has approved three new projects for Argentina, including a $300 million loan to improve health service quality and coverage.

A study has found that the lack of registration of some essential medicines in Kenya, Tanzania and Uganda is restricting access to safe and affordable healthcare. Simultaneously, over-registration of non-essential drugs is diverting regulatory resources towards non-priority treatments.

In the eastern part of the Democratic Republic of Congo, the WHO is warning that violence, natural disasters and disease outbreaks have brought the health system to a breaking point.

In Mozambique, the cholera outbreak has resulted in over 30,000 reported cases and over 130 deaths till the end of May.

In Iraq, prohibitively expensive healthcare is leading people to turn to natural remedies instead. 

In Afghanistan, Crimean-Congo haemorrhagic fever continues to spread, with over 90 new cases in the past week.