Sanofi and Sobi’s once-weekly haemophilia A treatment, Altuviiio, has been approved by the US Food and Drug Administration (FDA) for the prevention of and on-demand treatment to control bleeding episodes, as well as for surgery management for adults and children with the disease.

Haemophilia A patients lack sufficient functioning factor VIII protein to help their blood clot, causing external bleeding, bruising and bleeding into joints.

Current factor VIII therapies cause protein levels to increase but fall quickly, meaning patients usually have to go in for routine treatment every two days.

As Altuviiio has a half-life that is three to four times longer than standard and extended half-life factor VIII products, it allows for weekly dosing.

The FDA’s decision, which marks the first regulatory approval of Altuviiio, was supported by results from a phase 3 study in which the therapy demonstrated superiority to prior factor prophylaxis treatment.

Once-weekly Altuviiio prophylaxis met the study’s primary endpoint, providing significant bleed protection for people with severe haemophilia A with a mean annualised bleeding rate (ABR) of 0.7 and a median ABR of zero.

The treatment also met the key secondary endpoint of the study, with a significant reduction of 77% in ABR versus prior factor prophylaxis based on an intra-patient comparison.

Moderna and Life Edit Therapeutics, an ElevateBio company, have entered into a strategic collaboration to discover and develop in vivo mRNA gene editing therapies for hard-to-treat diseases.

The partnership will combine Life Edit’s suite of gene editing technologies with Moderna’s mRNA platform to advance therapies against a select set of therapeutic targets.

Under the terms of the agreement, both companies will collaborate on research and preclinical studies funded by Moderna and, if it chooses to exercise its optioning rights for these targets, the mRNA company will assume responsibility for further development, manufacturing and commercialisation.

While specific financial details have not been disclosed, Life Edit will receive an upfront payment from Moderna and will be eligible for certain milestone payments for each selected target, as well as tiered royalties on global net sales.

Life Edit’s gene editing platform includes a large library of base editors and RNA-guided nucleases, which are smaller in size when compared to conventional nucleases, potentially allowing for greater versatility for delivery.

Moderna’s partnership with Life Edit is not the company’s first of the year, after it announced a partnership in January with cancer-focused drug developer CytomX Therapeutics to create investigational mRNA-based conditionally-activated therapies for a wide range of diseases.

The US Food and Drug Administration’s (FDA’s) panel of advisors has unanimously voted in favour of an over-the-counter (OTC) version of Emergent BioSolutions’ opioid overdose-reversing drug.

If approved, Narcan (naloxone HCl) nasal spray will be the first opioid overdose medicine to be sold OTC nationwide, significantly expanding access to the life-saving treatment.

Opioids are commonly used for the treatment of pain and include medicines such as morphine, fentanyl and tramadol.

However, due to their pharmacological effects, the drugs can cause breathing difficulties and opioid overdose can lead to death.

The number of overdoses has increased in recent years in several countries, partly due to the increased use of opioids in the management of chronic pain and increasing use of highly potent opioids appearing on the illegal drug market.

Narcan, which was developed to be used at home without the need for any medical training, works by blocking the effects of an opioid overdose if administered in time.  However, it is currently only available in the US as a prescription medicine.

While not obligated to do so, the FDA typically follows the recommendations of its expert panel and is expected to make its final decision on prescription-free sale of Narcan by 29 March.

Ipsen will acquire exclusive worldwide rights to a pre-clinical stage programme with potential oncology applications, the company and its two partners, the Université de Montréal and IRICoR, announced.

Under the terms of the agreement, the Université de Montréal will receive an upfront payment from Ipsen and be eligible for development and commercial milestones, as well as royalties on net sales.

The agreement comes as a result of the collaboration established in May 2020 between the three partners, who said they are expanding their partnership with a new multi-year collaboration and option agreement for two discovery-stage oncology programmes.

As part of the new deal, a team of from the Institute for Research in Immunology and Cancer at the Université de Montréal will be responsible for the discovery and advancement of therapeutic compounds up to the drug candidate stage.

In exchange, Ipsen will pay the Université de Montréal an upfront fee and research funding for two compounds that reach the drug candidate stage and, should the company decide to exercise the option, it would assume all global development and commercialisation responsibilities of resulting candidates.

In this case, the Université de Montréal would also receive further milestone payments and be entitled to royalties for any commercialised product.