Tagged with Rare Diseases

Italy Speaking exclusively to PharmaBoardroom, Pierluigi Antonelli outlines Angelini Pharma’s progress in transforming from a traditional, Italy-focused, and family-owned firm into a true European innovator. Antonelli outlines the potential impact of Angelini’s USD 960 million Arvelle acquisition, why he is optimistic about success in the challenging brain health space, and how…

Pharma Legal Handbook The key facts about orphan drugs & rare diseases in Brazil. Prepared in association with Trench, Rossi e Watanabe one of Brazil’s most prestigious law firms, this is an extract from The Pharma Legal Handbook: Brazil, which can be purchased for GBP 119, here.   1. What is the definition of Rare Diseases…

Italy Claudia Coscia outlines how the geographic and portfolio transformation of Kyowa Kirin has played out in Italy in recent years. Coscia also touches on access challenges for rare disease therapies in the Italian pharma market, rising levels of rare disease awareness, and the digitalisation status of the country’s healthcare system. …

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…

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…