Four decades of orphan drugs and priorities for the future

6 July 2024 - The Orphan Drug Act was enacted in the United States in 1983 in response to growing awareness ...

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Issues, challenges and opportunities for economic evaluations of orphan drugs in rare diseases: an umbrella review

25 May 2024 - There are significant challenges when obtaining clinical and economic evidence for health technology assessments of rare diseases. ...

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Setmelanotide acetate for the treatment of patients with obesity and hyperphagia in Bardet-Biedl syndrome (final guidance)

22 May 2024 - NICE has published final evidence-based recommendations on the use of setmelanotide acetate (Imcivree) for treating obesity ...

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PHARMAC shares the agenda items for the upcoming Rare Disorders Committee meeting (May 2024)

13 May 2024 - PHARMAC is sharing what medicine applications will be considered at the Rare Disorders Advisory Committee meeting ...

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FDA grants Medivir´s MIV-711 rare paediatric disease designation and orphan drug designation for the treatment of Legg-Calvé-Perthes Disease

25 April 2024 - Medivir announced today that its selective cathepsin K inhibitor, MIV-711, has been granted rare paediatric disease designation ...

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Stealth BioTherapeutics announces FDA acceptance of new drug application for elamipretide for the treatment of Barth syndrome

8 April 2024 - If approved, elamipretide would become the first approved therapy for Barth syndrome. ...

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Collaborating to help improve access to drugs for rare diseases

29 February 2024 - On Rare Disease Day — a day when the international community raises awareness about rare conditions ...

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Today is Rare Disease Day

29 February 2024 - PHARMAC understands that people living with rare disorders face many challenges, including access to health care ...

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European Commission approves first CRISPR/Cas9 gene-edited therapy, Casgevy (exagamglogene autotemcel), for the treatment of sickle cell disease and transfusion-dependent beta thalassaemia

13 February 2024 - Over 8,000 patients 12 years of age and older with severe sickle cell disease or transfusion-dependent ...

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Rhythm Pharmaceuticals announces positive reimbursement decision in Italy for Imcivree (setmelanotide) for the treatment of obesity and control of hunger in Bardet-Biedl syndrome

7 February 2024 - Rhythm Pharmaceuticals today announced that the Italian Medicine Agency (AIFA) approved reimbursement (Gazzetta Ufficiale) for Imcivree (setmelanotide) ...

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New orphan drug for a sub-type of acute myeloid leukaemia: G-BA sees significant additional benefit

18 January 2024 - Since July 2023, there has been one in Germany for patients who suffer from acute myeloid leukaemia ...

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Sebelipase alfa for the treatment of patients with Wolman disease (final guidance)

10 January 2023 - Sebelipase alfa is recommended as an option for the treatment of patients with Wolman disease only if ...

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Orphan drug label expansions: analysis of subsequent rare and common indication approvals

8 January 2024 - We found that 491 novel orphan drugs were approved between 1990 and 2022.  ...

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Velmanase alfa for the treatment of patients with alfa mannosidosis

13 December 2023 - NICE has published final evidence-based recommendations on the use of velmanase alfa (Lamzede) for treatment of ...

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Cost of exempting sole orphan drugs from Medicare negotiation

27 November 2023 - This cross-sectional study identified 25 “sole orphan” drugs qualifying for exemption from Medicare price negotiation.  ...

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