Tagged with orphan drugs

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…

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,…

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…

GW Pharma Founded in 1998, GW Pharmaceuticals is a British biopharmaceutical company, best known for being the only company with a license to cultivate cannabis in the UK. True pioneers, they are the producers of Sativex and Epiodiolex, the former being the world’s first prescription medicine derived from the cannabis plant —…

USA 80 orphan drugs (those drugs intended to treat diseases so rare that sponsors are reluctant to develop them under usual marketing conditions) were approved in the USA in 2017, a record year and more than double the 40 approved in 2016.   Orphan drug spending grew from 5 to 10%…

India Anay Shukla leads the pharmaceutical, life sciences, med-tech and healthcare practice at Nishith Desai Associates, a leading Indian law firm. In this article, he examines the benefit of the recent policy changes to price controls for innovative drugs in India.  Unlike many other countries, India controls prices of all drugs…

Europe 125 medicines for rare diseases were approved by the EMA between 2004 and 2014, of which 71 were granted orphan drug status. This chart shows the availability of 112 of these drugs across European countries. The largest number of medicines for rare diseases were in continuous use in Germany and…

Turkey Abidin Gulmus, founder and chairman of Gen Ilaç, describes the company’s impressive journey to become Turkey’s principle orphan drug distributor and shares his vision for leveraging the company’s domestic expertise to expand their reach into foreign markets.   Gen Ilaç is one of the fastest growing Turkish success stories although…

Korea Korean Pharma company, Hanmi has won orphan drug status this week from the US Food and Drug Administration (FDA) for HM43239, a drug for the treatment of acute myeloid leukaemia (AML).   The endorsement of FDA’s Orphan Drug Designation (ODD) — invented to smooth the development of therapies for life-threatening…