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orphan diseases

This page shows the latest orphan diseases news and features for those working in and with pharma, biotech and healthcare.

EC approves Atara and Pierre Fabre’s Ebvallo as monotherapy for Epstein-Barr patients

We are proud and excited to bring this innovative therapy to the marketplace, which will reinforce Pierre Fabre’s portfolio in oncology, haematology and rare diseases.”. ... Ebvallo has orphan designation in Europe, which is reserved for therapies

Latest news

FDA awards over $38m for advancement of rare disease treatments

The grants and contracts, which were funded by the FDA’s Orphan Products Grants Program, will support clinical trials, natural history studies and regulatory science tools related to rare diseases. ... Additionally, the two FDA funded contracts are
PMEA 2020: continuing to recognise excellence in improving patient outcomes

Excellence in Rare Diseases and Orphan Drugs categories. ... with support from Learner Adams Bones Ltd. 8. Excellence in Rare Diseases and Orphan Drugs.
Building a brighter future for rare disease patients

PMC is proud to work with several orphan diseases organisations with innovative technology platforms and support their medical affairs efforts. ... The calibre of the shortlist in the Excellence in Rare Diseases, and Orphan Drugs category at the PMEA
Raising awareness and encouraging support on Rare Disease Day 2020

These include:. New requests for applications for the Orphan Products Grants programme, which supports natural history studies and clinical trials for rare diseases. ... Raising awareness is central to our mission to improve outcomes for people affected
Biotech now vital to pipelines – but success rates falling

Among these drugs, 27% were treatments for cancer and its symptoms and 20% are for the treatment of infectious diseases. ... Almost half had orphan drug status and over a third were identified by the FDA as first-in class therapies – such as

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Latest Intelligence

Listen, learn and lift off

The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) are expecting a busy year for approvals, with frantic activity in orphan diseases as gene and cell therapy ... That has completely changed and we’re now launching
Piloting a route to the biosimilars opportunities in Europe

Biological medicines represent a major advance in the treatment of serious pathological conditions such as cancer, neurodegenerative and autoimmune diseases, in the form of biosimilars. ... For orphan designations (medicine for rare diseases),
Assessing the impact of disease prevalence on access and pricing of rare disease drugs in England

Comparison of orphan drug assessments from NICE versus the NHS. The question of how to adequately capture the holistic value of orphan drugs (ODs) for rare diseases is the subject of ... to drugs targeting ‘very rare’ diseases (typically ultra-orphan
Biopharma and orphan drugs

Rare disease regulations. Twenty one of the 48 novel drugs approved in the US were for rare or orphan diseases – those that affect fewer than 200, 000 citizens. ... Clinical complications. Given the rarity of orphan diseases, one of the greatest
Industry needs an aligned position on the future of the EU OMP Regulation

The Pharmaceutical Strategy is likely to include innovation-boosting measures and plans to revise the EU Regulation on Orphan Medicinal Products (OMP Regulation). ... of orphan diseases).

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Latest appointments

Amryt Pharma appoints Jordi Casals as head of Europe

He joins the company from Aegerion Pharmaceuticals. Amryt Pharma, a company focused on treatments for rare and orphan diseases, has appointed Jordi Casals as the biopharma’s head of Europe.
Sobi appoints Norbert Oppitz SVP, speciality care

He joins the rare disease pharma firm from BSN Medical. Rare diseases healthcare company Swedish Orphan Biovitrum (Sobi) has appointed Norbert Oppitz as its senior vice president for its newly established
Minoryx Therapeutics adds to board of directors

He brings over 30 years of regulatory strategy expertise to the orphan diseases specialist, and currently also serves as a board member at Sarepta Therapeutics.
Minoyrx Therapeutics appoints chief medical officer

Dr Uwe Meya joins from Lupin Atlantis Holdings. Spanish orphan disease specialist Minoryx Therapeutics has appointed Dr Uwe Meya as its new chief medical officer. ... He will also support the further development of our pipeline in orphan diseases.”.
Ovid Therapeutics adds member to scientific advisory board

Ovid is a privately-held biopharmaceutical company focused on developing therapies for rare and orphan diseases of the brain. ... He said: “Ovid is pursuing an ambitious approach of tackling rare neurological diseases with significant unmet needs.

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Latest from PMHub

Rare and Orphan Diseases

1. It’s also predicted that by 2026 orphan drugs will make up almost 1/3 of the global drug pipeline’s value. ... Hadjivasiliou, A. and Senior, M. ‘Evaluate Orphan Drug Report 2022’. Available at: (Date accessed: January 2023).
Rare Diseases - not as rare as you think

There are more than 7000 rare diseases affecting an estimated 3.5 million people in the UK. ... The European Medicines Agency (EMA) supports the development and authorisation of medicines for rare diseases (often referred to as ‘orphan’ diseases)
Onyx Health Joins Forces with Rare Disease Specialists Actigen

Actigen's work focuses on identifying and developing biological medicines for rare diseases with unmet medical needs. ... At Actigen, our mission is to improve the lives of individuals with rare diseases, and we hope that GNR-055 will be the start of
How to Simplify Genetics & Raise Awareness Around Orphan Diseases

Gail Ouellette, President and Scientific Director of Regroupement Québécois des Maladies Orphelines ( RQMO)/Quebec Coalition of Orphan Diseases and Creator of the “ Genetics Simply" website, shares her thoughts on the barriers ... faced by rare
Lucid Group has four programmes shortlisted for PMEA 2019

Category: Excellence in Innovation. DermDialogue: Bringing innovation to MSL‐led education. Excellence in Rare Diseases and Orphan Drugs.