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FDA and NICE back Milton Park innovators
Published on 28 March 2025
Image caption: JOURNAVX bottle and tablet (photo: Vertex Pharmaceuticals / Business Wire)
From fighting cancer to tackling pain, several Milton Park occupiers have received milestone approvals from the US Food & Drug Administration (FDA) and National Institute for Health and Care Excellence (NICE) over recent months.
The breakthroughs showcase how Milton Park occupiers are developing groundbreaking therapies that improve health outcomes not just here in the UK, but across the globe.
Here are some recent announcements from Milton Park-based companies:
- Adaptimmune receives FDA designation for rare sarcoma therapy
- Immunocore secures FDA and NICE approval for its KIMMTRAK eye cancer treatment — which was recently fast-tracked to hundreds of patients across England
- Replimune secures priority review for advanced melanoma treatment
- Vertex Pharmaceuticals receives FDA approval for pain signal inhibitor
Adaptimmune receives designation for rare sarcoma therapy
Following a successful Phase II trial, Milton Park-based Adaptimmune has received a Breakthrough Therapy Designation for lete-cel, a T-cell therapy used to treat synovial sarcoma, a rare form of cancer which occurs near large joints.
Following the designation, the therapy will benefit from additional guidance from the FDA, with the potential for a priority review and other opportunities to speed up the development.
Adrian Rawcliffe, Chief Executive Officer at Adaptimmune, commented: “This designation by the FDA highlights the potential of lete-cel to address a critical need for new treatment options for patients with MRCLS. This is another important milestone in building out our sarcoma franchise, as we aim to bring Lete-cel to market in 2026 for the treatment of synovial sarcoma and MRCLS.”
Immunocore secures FDA and NICE approval for eye cancer treatment
Park occupier, Immunocore has achieved a historic first following FDA approval of KIMMTRAK (tebentafusp), a treatment option for patients with metastatic uveal melanoma (mUM), an aggressive form of eye cancer.
The treatment, which has also been approved by the National Institute for Health and Care Excellence (NICE), has been fast-tracked to hundreds of patients by the NHS across England and described by a patient as “the best early Christmas present I could’ve asked for”.
Professor Peter Johnson, NHS National Clinical Director for Cancer, said: “This type of melanoma is difficult to treat when it has spread in the body, so it is great news that the NHS can now offer this pioneering treatment, giving people an option that can extend their lives and offer them valuable extra time with their families and friends.
Bahija Jallal, Chief Executive Officer of Immunocore, commented: “Every year, hundreds of people are diagnosed with metastatic uveal melanoma who, until now, had no approved treatment options.
“KIMMTRAK is the first therapy to demonstrate a survival benefit to patients with this disease and we are focused on making KIMMTRAK available as quickly as possible.”
Replimune secures priority review for advanced melanoma treatment
Replimune has secured FDA priority review and a Biologics License Application acceptance for RP1, a treatment offering new hope for patients with a form of advanced melanoma (skin cancer).
The treatment is based on a proprietary strain of herpes simplex virus engineered with a protein intended to maximise tumour killing potency and tumour cell death.
The FDA grants priority review to applications for medicines that prove significant improvements in the safety or effectiveness of the treatment of a serious condition.
Sushil Patel, Ph.D., Chief Executive Officer at Replimune, commented: “There are limited treatment options and a significant unmet need for patients with advanced melanoma who previously received an anti-PD-1 containing regimen.
“The Biologics License Application acceptance is an important milestone for Replimune and we look forward to working closely with the FDA on the review of our application.”
Vertex Pharmaceuticals receives FDA approval for pain signal inhibitor
Milton Park-based Vertex Pharmaceuticals has secured FDA approval for JOURNAVX, the first non-opioid oral pain signal inhibitor in over 20 years for use across all types of moderate to severe acute pain. The medicine offers an effective, well-tolerated alternative to opioids without the risk of addiction as seen in other opioid derivatives such as morphine. The drug was developed in the R&D labs here at Milton Park.
Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex, commented: “A historic milestone for the 80 million people in America alone who are prescribed a medicine for moderate-to-severe acute pain each year.
“With the approval of JOURNAVX, a non-opioid, pain signal inhibitor and the first new class of pain medicine approved in more than 20 years, we have the opportunity to change the paradigm of acute pain management and establish a new standard of care.”
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