Three investigational therapies targeting severe rare diseases have received the first Innovation Passports under the UK's newly enhanced Innovative Licensing and Access Pathway (ILAP), marking a significant milestone in accelerating access to transformative medicines for NHS patients. The recipients include treatments for life-threatening conditions where current therapeutic options are extremely limited or non-existent.
The ILAP Partners—comprising the Medicines and Healthcare products Regulatory Agency (MHRA), National Health Service (NHS), and health technology assessment bodies including NICE, the Scottish Medicines Consortium (SMC), and the All Wales Therapeutics and Toxicology Centre (AWTTC)—announced the selection from 16 applications in the first round.
World-First Collaborative Approach
ILAP distinguishes itself as the only end-to-end pathway globally where healthcare developers, regulators, the UK-wide national health system, and health technology assessment bodies work together from the outset. The pathway provides developers with early, coordinated guidance on safety, effectiveness, and value requirements across the entire system, designed to reduce the time required for promising new treatments to progress through development, licensing, and ultimately reach NHS patients.
The enhanced ILAP builds on the original pathway launched in 2021, incorporating key improvements based on feedback and successes. The new version focuses more selectively on transformative products addressing unmet clinical needs, with NHS partners now embedded as core members to ensure faster system-wide adoption. Enhanced features include bespoke support, predictable timelines, early patient and NHS engagement, and a single point of contact for each product.
Addressing Critical Rare Disease Needs
The three selected investigational products target conditions with significant unmet medical needs:
Neonatal onset ornithine transcarbamylase (OTC) deficiency: A rare metabolic disorder that prevents the body from breaking down ammonia, often proving fatal in infancy without liver transplantation. iECURE's investigational therapy ECUR-506 utilizes an innovative targeted gene insertion platform to address this severe condition.
Duchenne muscular dystrophy: A progressive muscle-wasting disease affecting 1 in 3,500-5,000 boys, currently with no cure available.
Rare pediatric neurodegenerative condition: Affecting children with no current cure or treatment options.
The rare disease landscape presents particular challenges for drug development. Between 5,000 and 8,000 rare diseases exist, each affecting less than 0.1% of the UK population, but collectively impacting 3 million people. For many of these conditions, effective treatments remain lacking, leaving patients and families with limited options. Small patient populations make clinical trials complex and expensive, creating significant barriers to development.
Industry and Regulatory Support
Lawrence Tallon, Chief Executive of the MHRA, emphasized the collaborative nature of the initiative: "The new ILAP is collaborative regulation at its best – patients, healthcare partners, industry, and regulators working together to deliver meaningful benefits for public health. By embedding NHS partners from the start and focusing on transformative medicines, this world-leading, integrated pathway keeps the UK at the forefront of innovative medicine and helps overcome some of the barriers in rare disease development."
Dr Sam Roberts, Chief Executive of NICE, highlighted the pathway's potential impact: "As an organisation committed to getting the best care to people fast, NICE welcomes any initiative that helps developers get transformative medicines into the NHS. The collaboration between partner organisations, industry and patients has really helped shape the new ILAP offer."
Joe Truitt, Chief Executive Officer of iECURE, expressed appreciation for the recognition: "We are honoured that iECURE's investigational therapy, ECUR-506, has been awarded an Innovation Passport to enter the ILAP. This unique initiative is designed to accelerate patient access to transformative new medicines, and we're proud to be part of that effort."
Pathway Forward
The Innovation Passport provides developers with coordinated support from the earliest development stages, including priority access to clinical trials support and NHS engagement. This comprehensive approach aims to speed up access for patients where current treatment options are limited or non-existent.
Entry to ILAP remains open to commercial and non-commercial developers worldwide, with applicants required to submit medicines or drug-device combinations that have not yet entered their confirmatory trial. This timing allows maximum benefit from the pathway's support offerings.
The selection of these three investigational products supports broader commitments outlined in the government's Rare Diseases Action Plan, which aims to improve diagnosis, treatment, and care for people living with rare conditions. The initiative also aligns with the ambitions of the 10 Year Health Plan for England and the Life Sciences Sector Plan, positioning the UK to harness research and innovation for improved patient outcomes while maintaining its global leadership in life sciences.
While these products remain in development, their inclusion in ILAP represents hope for families currently facing limited treatment options. Future availability will depend on demonstrated evidence of safety, effectiveness, and value through the development process.
