The MHRA just did something significant for rare diseases
The MHRA has launched a public consultation on a proposed new Rare Disease Therapies Regulatory Framework, a new approach to how rare disease therapies could be tested, licensed, and monitored in the UK.
The headline change is a new Investigational Marketing Authorisation, which would combine clinical trial approval with a progressive route to market authorisation. This means patients could gain access to innovative treatments earlier, where evidence is limited, subject to NICE approval for NHS use.
The framework would apply to conditions affecting no more than 1 in 50,000 people, where conducting a standard clinical trial isn't feasible. For CGT developers working in ultra-rare indications, this is worth paying close attention to. The consultation closes 20 July and the MHRA wants to hear from developers, clinicians, and patient groups.
Read more: Gov.uk
The MHRA also approved the UK's first genetic medicine for a rare skin condition
Separately, the MHRA granted marketing authorisation to Vyjuvek (beremagene geperpavec) from Krystal Biotech for the treatment of wounds in patients with dystrophic epidermolysis bullosa, making it the first genetic medicine approved in the UK for DEB.
Vyjuvek works by copying a functional version of the faulty gene into wound cells to support healing. In a Phase III study of 31 patients, 67% of wounds healed completely at six months, compared to 22% in the placebo arm.
The approval allows for flexible administration, including dosing at home by patients or caregivers, and covers patients from birth. This is Krystal's fourth approval globally, following the US, EU, and Japan. A clean example of what global regulatory momentum looks like when it builds.
Read more: Precision Medicine Online, European Pharmaceutical Review GlobeNewswire
A first-in-human CAR-T trial just opened for multiple myeloma
HaemaLogiX has opened patient enrolment for its Phase 1 KOALA trial at the Peter MacCallum Cancer Centre in Australia, the first human study of its KMCAR T-cell therapy for relapsed or refractory kappa-restricted multiple myeloma.
What makes this one worth watching: KMCAR targets the Kappa Myeloma Antigen, which is found on myeloma cells but not on healthy immune cells, a selectivity that, in theory, could allow effective tumour killing while preserving normal immune function. That would set it apart from currently approved BCMA-directed CAR-T approaches.
The dose-escalation trial will assess safety and early efficacy, following TGA approval and ethics clearance in Australia. Early days but a differentiated target and a credible preclinical story make this one to follow.
Read more: Pharmatimes
See you next Friday.
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