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European Commission licenses first treatment for acanthamoeba keratitis
Following its European approval, the small molecule treatment is set to become the standard of care for patients with the ultra-rare disease.
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Oligonucleotide ALS therapy approved in EU
The European Commission (EC) has granted Biogen its third rare disease treatment approval in the European Union.
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Why patient-centricity is essential to accelerate rare disease clinical research
There is an urgent need for novel treatment options for people living with rare diseases, but carrying out clinical research in this field is notoriously difficult. As Clinical Trials Day approaches, Cezary Statuch, Head of Medical Affairs International and Japan at Alexion Pharmaceuticals, highlights how adopting patient-centric approaches can help…
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EU pharmaceutical legislation revisions: what are the implications for biopharma?
Anne Dhulesia and Sean Dyson, Partners at L.E.K. Consulting, discuss the proposed revisions to EU pharma legislation and potential implications for biopharma companies operating in Europe.
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CHMP meeting highlights – May 2023
The Committee for Medicinal Products for Human Use (CHMP) recommended two new medicines for approval in its May meeting, including a neurosteroid for epileptic seizures.
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EMA’s human medicines committee (CHMP) meeting highlights – February 2023
In its latest meeting, the Committee for Medicinal Products for Human Use (CHMP) recommended eight new medicines for approval, including an enzyme replacement therapy for a rare disease.
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Therapeutic-first for cold agglutinin disease haemolytic anaemia
The European Commission has approved Enjaymo®, the first-and-only approved therapeutic for haemolytic anaemia in adults with cold agglutinin disease.
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EMA human medicines committee (CHMP) highlights, November 2022
The Committee for Medicinal Products for Human Use (CHMP)’s November meeting consisted of approval recommendations for four medicines, including a biosimilar for osteoporosis and a new COVID-19 vaccine.
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$38 million will fund rare disease research over four-year period
Research for rare diseases including ALS will be supported over the next four years by $38 million in FDA funding.
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RemeGen receives ODD for myasthenia gravis treatment
The FDA has granted ODD to Telitacicept, produced by RemeGen, for the treatment of myasthenia gravis.
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Serplulimab increases survival in small cell lung cancer patients
Hansizhuang (serplulimab), the world's first anti-PD-1 monoclonal antibody (mAb) treatment for extensive-stage small cell lung cancer (ES-SCLC), enhanced survival rates compared to chemotherapy, and is projected to be the leading immunotherapy for the disease in the next five years.
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CAR-T therapy safe for relapsed or refractory T-cell malignancies, finds study
A Phase I trial revealed that Bioheng Biotech’s chimeric antigen receptor (CAR) T-cell therapy drug RD13-01 was safe for use and that it could reduce malignant tumours.
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Using RWE in rare disease drug development: effective innovations with historical controls
With a growing number of therapies under development for rare diseases, ICON’s Dr William Maier discusses how real-world evidence (RWE) can be effectively used as a historical control (HC), overcoming challenges presented in clinical development.
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EMA human medicines committee (CHMP) highlights, September 2022
In its latest meeting, the Committee for Medicinal Products for Human Use (CHMP) recommended 12 medicines for approval, including those for rare diseases, osteoporosis and cancer.
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Europe approves first gene therapy for treatment of haemophilia A
European approval of BioMarin’s ROCTAVIAN (valoctocogene roxaparvovec) one-time gene therapy represents a breakthrough in the treatment of adults with severe haemophilia A.
