Tagged with Rare Disease

Spain Sobi’s VP and general manager for Spain and Portugal, Pablo De Mora, comments on his experience during the pandemic, the Swedish company’s haematology and immunology portfolio, including its EMA-approved COVID-19 treatment, and the reimbursement challenges faced by rare disease players in Spain. Moreover, De Mora highlights Sobi’s vast clinical trials…

Spain The Spanish Association of Orphan and Ultra Orphan Medicinal Products Laboratories (AELMHU) is a non-profit organisation working to improve awareness about rare diseases and access of Orphan Drugs. Maria Jose Sanchez Losada, president of the association, comments on the current status of diagnosis, access and reimbursement of rare diseases and…

Saudi Arabia The general manager for Recordati Rare Diseases in MENA, Tony Zbeidy, explains why awareness of rare disorders has vastly improved in the region, which has one of the largest incidence rates in the world, the particular importance of the Saudi Arabian market to Recordati, the need for a more collaborative…

Saudi Arabia Jean Paul Scheuer, General Manager for Sanofi Genzyme and MCO Lead for Greater Gulf, highlights the company’s expanding footprint in the region, the organizational changes brought about by global CEO Paul Hudson, and how Sanofi has managed to launch 20 new products in the last year. Sanofi has been quick…

Singapore Writing in the December edition of DIA’s Global Forum magazine, Agnes Ho, Cindy Zen, and Tan Kwee Lan of Boehringer Ingelheim give an overview of the current regulatory framework for orphan drug registration in four ASEAN countries and what more could be done to improve it.   Regulatory authorities such…

Saudi Arabia Myriam Hakim outlines the progress that Japanese rare disease and oncology specialist Kyowa Kirin has made in the GCC region in recent years; the importance of the Saudi market to Kyowa Kirin and its customised strategy there; as well as the main game-changers for patients in the region, namely the…

Saudi Arabia Few stakeholders are better placed to assess the landscape for rare disease therapies across the Middle East and North Africa (MENA) region than Karim Smaira and Kamel Ghammachi of Genpharm, an entrepreneurial success story that now provides market access and marketing solutions for rare disease products across the region.  …

Saudi Arabia Ipsen’s general manager for Middle East and Africa (MEA), Khaled Elrefae, highlights the French firm’s expanding footprint in the region, the fundamentals that make MEA a growth driver for Ipsen, and the opportunities for further growth from its oncology, rare disease and neuroscience portfolios. In addition, Elrefae discusses the changing…

Turkey Ercin Kugu, CSL Behring’s general manager for Turkey and Israel, highlights the rare disease company’s footprint in Turkey, explains the different regulatory framework in the country for plasma products, and provides insights on the process of establishing Big Pharma affiliates in Turkey from zero after doing so for both Shire…

USA The National Organization for Rare Disorders (NORD) is set to host its annual Rare Diseases and Orphan Products Breakthrough Summit on October 18-19th. Ahead of the Summit, the Chair of NORD’s Board of Directors Kay Holcombe explains its significance for cross-stakeholder interaction and learning and why we are entering “a…

Saudi Arabia Kamel Ghammachi and Karim Smaira are the co-founders of Genpharm, an entrepreneurial success story that now provides market access and marketing solutions for rare disease products across the MENA region. They discuss the impact of COVID-19 on their business; the evolving dynamics of the Saudi pharma market; and the crucial…

Turkey Few Turkish companies are experiencing such a transformational moment as GEN. Just in 2021, the Ankara-based organization made its public debut at the Istanbul Stock Exchange and signed an exclusive collaboration agreement with a Dutch biotech company for the development and commercialization of a therapy against Alzheimer’s and other neurodegenerative…