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rare diseases

Roche scores two EU approvals in haemophilia and rare autoimmune disease

The European Commission (EC) has approved Roche’s haemophilia treatment Hemlibra (emicizumab).

The approval comes as the EC approved Roche's MabThera (rituximab) for the rare autoimmune disease pemphigus vulgaris (PV) marking the first major advancement in treatment fo the disease in more than 60 years.

Hemlibra, a bispecific antibody targeting factor IXa- and factor X was approved as a treatment in routine prophylaxis of bleeding episodes in severe haemophilia A patients without factor VIII inhibitors.

China to establish national rare disease network

China is set to establish a national collaborative network of hospitals for rare disease diagnosis and treatment to promote early detection and effective treatment of rare diseases.

The initiative, announced by the National Health Commission, will see 324 hospitals, chosen for their capacity and experience in treating patients with rare disease, participate in the network.

The network will facilitate the timely transfer of difficult and complicated cases between hospitals and the allocation of quality medical resources for patients.

The Autumn 2018 edition of Pharmafile is available to read online now!

The Autumn 2018 edition of Pharmafile, our long-running bi-annual guide to the latest trends, projections and thought leadership positions in the pharmaceutical and life sciences industries, is now available to read online.

In this new edition of our revamped format, Pharmafile centres on three specially selected therapeutic areas: infectious diseases, rare diseases, and immunology.

FDA approves AstraZeneca’s Lumoxiti for hairy cell leukaemia (HCL)

AstraZeneca’s Lumoxiti has been approved for use for the treatment of relapsed or refractory hairy cell leukemia (HCL)in adults who have had at least two prior therapies for the disease.

The FDA granted the CD22-directed cytotoxin Fast Track and Priority review designations as well as Orphan Drug designation which is intended to provide incentives to assist and encourage the development of drugs for rare diseases.

Global orphan and rare dermatology market set to exceed $6 billion by 2024

Image Credit: Rafael Matsunaga

The global orphan and rare dermatology premium products market is expected to grow rapidly in the next half a decade according to the business intelligence firm GBI Research.

The market is set to be worth $6.07 billion by 2024 having been valued at just $1.64 billion in 2017. The research published in GBI’s latest report ‘Global Orphan and Rare Dermatology Drugs Market to 2024’notes that the market is expected to grow at a rate of 20.5% each year.

FDA approves RNA therapy for rare genetic disease

The US Food and Drug Administration has approved the RNA-based therapy Onpattro, for the treatment of the rare genetic disease polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients.

The approval marks the first treatment for patients with polyneuropathy caused by hATTR, a rare, debilitating, genetic disease characterized by the build-up of abnormal amyloid protein in peripheral nerves, the heart and other organs.

The need for flexible appraisal of rare disease medicines

Published on 15/03/18 at 10:30am

Recent data has shown that England is, on average, slower than Germany, France, Spain and Italy in making orphan medicinal products available to patients. Joe Wiley, CEO of Amryt Pharma, discusses the need to ensure faster access to treatments for people with rare diseases.

All diseases are unfair, but rare diseases are the most unfair of them all.

Taking science and making people feel connected to it

Published on 21/08/17 at 11:07am

Owen Marks, Country Head for the Rare Disease Unit at Pfizer UK, discusses his career so far and what drives his work in rare diseases.

What made you pursue a career in the pharmaceutical industry?

SMi's 7th annual Orphan Drugs and Rare Diseases UK Conference

Shire’s drug picked up from Baxalta given fast track designation

Shire has announced that its drug, SHP655, has received a fast track designation based on pre-clinical trials and positive results from a Phase 1 study. The drug is a treatment for patients with hereditary thrombotic thrombocytopenic purpura (hTTP), a rare blood disorder 3,000 to 4,000 patients suffer from globally.

hTTP is a disorder in which individuals are congenitally deficient in ADAMTS13, an enzyme that cleaves the von Willebrand factor protein involved in blood clotting. The risk of death is high if the condition is left untreated.

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