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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.
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Orphan Drugs
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2022 spells change for EU pharmaceutical legislation
With almost every aspect of pharmaceutical regulation currently under review across Europe and the proposal for changes expected in December 2022, in this article, Maarten Meulenbelt, Partner at Sidley Austin LLP, discusses with European Pharmaceutical Review some of the more controversial amendments under consideration and emphasises why industry participation is…
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Orphan drug sector continues to experience rapid growth
According to a new report, the orphan drug market is growing more than twice as fast as the non-orphan market and, by 2026, orphan drug sales will account for 20 percent of all prescription drug sales.
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FDA approves Besremi to treat rare blood disease
The US Food and Drug Administration (FDA) approves Besremi (ropeginterferon alfa-2b-njft) injection to treat adults with polycythemia vera.
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FDA awards 11 grants for rare disease treatment developments
The FDA has awarded 11 grants equating to about $25 million of funding to clinical trials for the development of rare disease treatments.
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EMA’s human medicines committee (CHMP) September meeting highlights
In its most recent meeting, the CHMP recommended nine new medicines and concluded its review of Vaxzevria’s thrombosis risk.
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Mobocertinib approved as first oral therapy for EGFR Exon20+ NSCLC
FDA approves mobocertinib as the first oral therapy for patients with non-small cell lung cancer with EGFR Exon20 insertion mutations.
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Moderna announces first patient dosed in mRNA-3705 study
Moderna have announced that the first patient has been dosed in Phase I/II study of mRNA-3705 for methylmalonic acidemia (MMA).
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MHRA approves Epidyolex® (cannabidiol) for TSC seizure treatment
GW Pharmaceuticals received approval for Epidyolex®(cannabidiol) for the treatment of seizures linked to tuberous sclerosis complex (TSC) in the UK.
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EMA’s human medicine committee (CHMP) meeting highlights, July 2021
The Committee for Medicinal Products for Human Use (CHMP) recommended two medicines for approval and provided a negative opinion on Nouryant.
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Highlights of the EMA’s human medicine committee meeting, April 2021
The Committee for Medicinal Products for Human Use (CHMP) recommended eight drugs for approval and nine indication extensions.
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Microbiome biotherapeutic shows promise in acute graft-versus-host disease
MaaT013 was safe and generated complete or very good partial responses in a third of patients with steroid-refractory, gastrointestinal-predominant acute graft-versus-host disease.
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EMA’s transformative treatments of 2020
In this article, we summarise the trial findings for the European Medicines Agency’s 13 most transformative therapies of 2020.
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What impact does disease rarity have on rare and orphan drug pricing?
Bhavesh Patel, Cécile Matthews and Owen Male at CRA’s Life Sciences Practice assess the relationship between drug pricing for rare disease therapies and disease prevalence in Europe, taking a closer look at payer assessments of orphan drugs in Germany.
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FDA grants Orphan Drug Designation to two cancer treatments
The FDA has given small molecules APG-115 and APG-1252, two cancer treatments, Orphan Drug Designation.
