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- On 13 January 2025, Plusultra pharma UK Ltd is pleased to announce that the Scottish Medicines Consortium has approved the Orphan designated drug Hyftor Gel (Sirolimus) to treat children and adults from the age 6 years and older with Facial angifibromas associated with Tuberous Sclerosis Complex in Scotland on the NHS.
Affecting approximately 6 in every 100,000 of the Scottish population, Tuberous Sclerosis Complex is a complex genetic disease and associated facial angiofibroma is one of its big components. Among 10 people diagnosed with TSC, 8 or 9 will also have associated facial angiofibroma. Facial angiofibroma shows as small facial bumps made of blood vessels and fibrous tissue, focused centrally on the face in a symmetrical butterfly pattern, on the nose and on the cheeks. In the early phase, facial angiofibromas often show as red-coloured dilatation of blood vessels.
When such lesions become large, they can create trouble with breathing, eating, speaking, or vision. Also, they can cause recurrent bleeding and infection, leading to scarring and disfigurement. Although facial angiofibromas are benign (not cancer), they can have negative psychological impact on a patient and the family/caregiver, such as emotional distress and social isolation.¹
The SMC’s decision was based on results from two pivotal phase 3 trials, In the Hyftor clinical trials NPC-12G-1 and NPC-12G-2², there was reduction in the number, size, and redness of facial angiofibromas associated with tuberous sclerosis complex after 12 weeks (3 months) of treatment twice daily and sustained effects (up to one year of treatment).
Hyftor is approved in Japan, China, The United States of America and it was approved and launched in Europe and the UK in November 2023.³
Scottish Medicines Consortium: sirolimus (Hyftor)
References
- Tuberous Sclerosis Signs and Symptoms 2020.
- https://pubmed.ncbi.nlm.nih.gov/29800026/; https://pubmed.ncbi.nlm.nih.gov/32385845/
- Marketing authorisation in Great Britain was granted by the Medicines and Healthcare Products Regulatory Agency (MHRA) on Sep 1st 2023 (MHRA PLGB number 57572/0001). Authorisation was given through the European Commission (EC) reliance procedure after European Medicines Agency (EMA) approval. See https://www.ema.europa.eu/en/medicines/human/EPAR/hyftor.
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About Plusultra Pharma
As a research and development-based pharmaceutical company, we develop pharmaceuticals for unmet needs, such as orphan drugs, off-label medicines, and paediatric medicines. Before delivering new products, we always consider the needs of our customers.
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Plusultra pharma, as a group of Nobelpharma, has earnestly addressed the launch of medicines that other companies do not pursue, in order to fulfil the needs of society.
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“Critical but neglected – That is precisely why it must be created.”
Plusultra pharma GmbH and Plusultra pharma UK Ltd. were established in Duesseldorf, Germany, and Oxford, United Kingdom, respectively in 2020, as overseas subsidiaries in Europa regions of Nobelpharma Group. The headquarter of the group, Nobelpharma was founded in 2003 in Tokyo, Japan, to fulfill our mission of “Contributing to Society by Providing Critical but Neglected Pharmaceuticals and Medical Devices” that other companies do not pursue. We have since obtained approvals for the manufacturing and sale of new drugs in numbers on par with large pharmaceutical companies, and we have been providing these drugs to patients. We take much pride in the unique corporate philosophy that has made this possible.