Tagged with Rare Diseases

France Franck Cousserans, general manager of France and Belgium at Jazz Pharmaceuticals, highlights the company’s key products and the challenges of market access in France. Furthermore, he indicates the steps needed to grow in the future and how Jazz’s corporate culture is key to its success.   In the US, Jazz…

UK Formed in 2015, listed UK start-up Amryt targets rare diseases with high unmet medical need. With one commercial asset on the market, a strong pipeline of development assets and a commercial infrastructure across the EMEA region already in place, the company is aiming to expand rapidly.   We have expanded…

Spain Aurora Berra, general manager of Sobi Spain and Portugal, highlights her key priorities and the opportunities and challenges inherent in the fragmented Spanish healthcare system. Berra gives insight into the need for a bespoke rare disease focus and delivers advice to young women starting their journey in the healthcare industry.…

Opinion Rhenu Buller, an expert on disruptive technologies at business consultancy Frost & Sullivan’s Asia Pacific office, outlines the technological innovations currently driving the drug development pipelines of rare disease companies.   Recent advancements in the development of novel platforms for precise targeting of small molecules, gene therapy, stem cell therapy,…

Opinion Kevin Rufang Huang is the president and founder of the Chinese Organization for Rare Disorders (CORD). A rare disease patient himself, he is responsible for bringing“International Rare Disease Day” to China. Consequently, rare diseases now have much more visibility in the country. As an expert on the subject, Kevin breaks…

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…