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The European Medicines Agency (EMA) has published recommendations to increase communication and planning efforts in a bid to stop the current medicine shortages from becoming even worse.  

Within the 14-page document, published by an EMA special task force, are 10 recommendations of best practices that “marketing authorisation holders, wholesalers, distributors, and manufacturers can consider adopting to ensure continuity of medicinal product supply and reduce the impact of shortages”. 

Amongst the recommendations are calls for informing national competent authorities of impending shortages, creating shortage prevention protocols, designing multinational supply chains to increase resilience, and communication lines between stakeholders and supply chains. 

The EMA set up a task force in 2016 in light of issues concerning medicine availability and supply chain. The group has been looking into solutions for improving the continuity of supply of human and veterinary medicines across Europe. 

The guidance adds to a previous one in 2022 aimed at patient and healthcare professional organisations as EMA tackles the instability of medicine supplies.

Forge Biologics has joined the public-private collaboration, the Bespoke Gene Therapy Consortium (BGTC), to expedite the development and manufacture of new AAV [adeno-associated virus] gene therapies to treat patients with rare diseases. 

The BGTC is part of the accelerating medicines partnership (AMP) programme and is managed by the Foundation for the National Institutes of Health (FNIH). 

The AMP programme is a public-private partnership between a number of biopharmaceutical and life sciences companies, the US Food and Drug Administration (FDA) and the National Institutes of Health (NIH), along with non-profit and other organisations. 

The BGTC aims to expedite the development, manufacture and delivery of customised or “bespoke” gene therapies to treat rare diseases, including some which are too rare to be of commercial interest. 

The AMP BGTC will also facilitate cost-efficient vector production to increase access for patients genetic diseases.

Scribe Therapeutics has entered a strategic collaboration with Eli Lilly and Company subsidiary Prevail Therapeutics for accelerating in vivo CRISPR-based therapies to target the causes of serious neurological and neuromuscular diseases. 

Under the deal, Prevail obtains exclusive rights to use Scribe’s CRISPR X-Editing (XE) technologies to develop the medicines. 

The deal will see the integration of Scribe’s new CRISPR by Design approach and Prevail’s expertise in developing genetic medicines for neurological disorders for specific genetic targets. 

Scribe will receive $75m, including an upfront payment and equity investment via a convertible note. The company is also eligible for development and commercial milestone payments exceeding $1.5bn. 

Prevail will pay for research funding and tiered royalties on net future sales while Scribe will have the right to opt-in to co-fund and share profits in the US on a single collaboration programme from the latest partnership.

Dutch biopharmaceutical company Byondis has received a complete response letter (CRL) from the US Food and Drug Administration (FDA) regarding its biologics license application (BLA) for [vic-]trastuzumab duocarmazine (SYD985). 

The CRL states that the FDA is suspending its decision and requests additional information, which will need time and resources to consider that might extend beyond the present evaluation period. In July 2022, the US FDA accepted the company’s BLA for its investigational next-generation anti-HER2 antibody-drug conjugate, SYD985, to treat HER2-positive unresectable locally advanced or metastatic breast cancer (MBC) patients. 

The application was supported by the findings from the pivotal Phase III TULIP clinical trial. The trial was designed to compare SYD985 to the physician’s choice (PC) to treat pre-treated HER2-positive unresectable locally advanced or MBC patients.

BioNTech has ended its research collaboration with Matinas after its oral mRNA vaccine failed to demonstrate preclinical activity. 

Matinas announced the news in a May 10 statement. The in vivo study, conducted in mice, involved a phosphatidylserine-containing nano-formulation of mRNA supplied by BioNTech, distinct from traditional lipid nanocrystals (LNCs). Matinas said it developed this unique formulation to “handle the physical complexity and biological fragility of mRNA”.

Whilst the formulation had been successful in vitro, the oral administration in mice did not elicit activity. 

The news means the collaboration between the two companies lasted just over a year. In April 2022, BioNTech paid an upfront access fee to use Matinas’ LNC delivery platform technology. At the time, BioNTech was excited to see what the LNC platform could do for the oral delivery of mRNA therapeutics.