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Neurofibromatosis type 1 (NF1)

CHMP meeting highlights – May 2025

27 May 2025 | By Catherine Eckford (European Pharmaceutical Review)

The EMA’s committee has granted positive opinions for ten new treatments, including medicines for oncology indications and rare diseases.

close up of the hands of a group of business people piled together in the middle of a circle - idea of collaboration

article

Collaboration and integration to progress clinical research

6 August 2021 | By Hannah Balfour (European Pharmaceutical Review)

Here, Lucinda Cash-Gibson and Francesco Patalano discuss why the pharmaceutical industry is collaborating to develop protocols for patient-centric integrated platform trials and what the clinical research industry may look like in future.

person with large neurofibromas on their back

news

EU approves Koselugo for treatment of neurofibromatosis and plexiform neurofibromas

22 June 2021 | By Hannah Balfour (European Pharmaceutical Review)

The European approval of Koselugo was based on its ability to reduce inoperable plexiform neurofibromas in paediatric patients with neurofibromatosis type 1.

Cartoon of people in white coats looking at various oversized medicines, including tablets and liquids in various packages.

news

Highlights of the EMA’s human medicine committee meeting, April 2021

26 April 2021 | By Hannah Balfour (European Pharmaceutical Review)

The Committee for Medicinal Products for Human Use (CHMP) recommended eight drugs for approval and nine indication extensions.

news

Cabozantinib reduces tumour burden in Phase II neurofibromatosis trial

15 January 2021 | By Hannah Balfour (European Pharmaceutical Review)

Forty percent of neurofibromatosis type 1 patients enrolled in the trial had a >20 percent reduction in tumour burden with cabozantinib.

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Recommended

Neurofibromatosis type 1 (NF1)

CHMP meeting highlights – May 2025

Collaboration and integration to progress clinical research

EU approves Koselugo for treatment of neurofibromatosis and plexiform neurofibromas

Highlights of the EMA’s human medicine committee meeting, April 2021

Cabozantinib reduces tumour burden in Phase II neurofibromatosis trial