Tagged with Rare Diseases

Opinion Drug development in rare diseases is currently in a very exciting time. While much of the focus is on the scientific advances,  access to rare disease patients and well-designed clinical trials are also essential to evaluating new therapeutics. In this article, PJ Brooks, Program Director at the Office of Rare…

Korea Min-Young Kim, general manager at Ipsen Korea discusses her strategy to refocus the Korean affiliate away from the toxin market and towards oncology and rare diseases. She also documents Ipsen’s rapid local growth and increasing market share. Miss Kim also gives her assessment of the current drug pricing situation, as…

Rare Diseases Long regarded as a neglected backwater within the drug discovery landscape and the preserve of only a handful of niche players, the rare disease space has been undergoing an extraordinary turnaround in fortunes of late.   According to EvaluatePharma’s forecasts, orphan drug sales are set to grow 11 percent each…

Rare Diseases The 28th February 2019, marks the twelfth international Rare Disease Day. On and around this day hundreds of patient organisations from countries and regions across the globe will strive to raise awareness of some of the world’s rarest disease. For many of the rare diseases out there, we are yet to…

Ipsen Following on from Johnson & Johnson’s acquisition of Actelion in January 2017 and Takeda’s takeover of Shire two years later, pharma multinationals’ rush to snap up rare disease specialists continues with Ipsen’s proposed acquisition of Clementia Pharmaceuticals. Industry insiders predict that this trend is set to continue well into the…

South Korea This week the Korean Ministry of Food and Drug Safety announced that as of 12th March 2019 a revised narcotics bill will go into effect that will allow imports of medical cannabis for self-treatment but under strict conditions.   Until now, the import, export, manufacturing and sale of cannabis has been…

Top 10 The Top 10 bestselling orphan drugs in the USA in 2017. Celgene’s Revlimid (generic name lenolidamide) leads the way with sales of USD 5.4 billion, followed by Roche’s Rituxan (rituximab) with USD 4.2 billion in sales and TEVA’s Copaxone (glatiramer acetate) with USD 3.1 billion. The most expensive drug in…

Top 10 The top 10 companies in the world ranked by global orphan drug sales and market share in 2017. Swiss giants Novartis and Roche take the top two places, followed by American firm, Celgene. Made with Visme Infographic Maker

Rare Diseases In the run-up to Rare Disease Day 2019, David H. Crean, PhD, Managing Director for Objective Capital Partners, reviews the investment and deal activity for orphan drugs in 2018 and delves into future forecasts for the rare disease space.   With drugs currently available for only about 5% of rare diseases,…

Canada Durhane Wong-Rieger is the President & CEO of the Canadian Organization for Rare Disorders (CORD). In her first article for PharmaBoardroom, Durhane explains the changing landscape for access to rare disease drugs in Canada and the actors involved in getting there.   The irony of the government processes, which, on…

Opinion After her son was diagnosed with SYNGAP1 – an extremely rare gene mutation – Monica Weldon founded Bridge the Gap – SYNGAP Education and Research Foundation to support families of those suffering from the illness and to accelerate the path to better therapies. Here, Monica shares her organisations secret to success in the…

China It has been announced by China’s State Council that as of March 1st 2019, the government will reduce the VAT on a “first batch” of 21 rare disease drugs and four active pharmaceutical ingredients (API’s) by more than 80% in a bid to get new treatments to market faster. Historically, China has…