Tagged with Orphan

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…

Innovation Patient groups oftentimes serve as the authority on a specific disease when it comes to advancing research.   “Without patient perspective, there is no good medical product development,” Rachel Sherman, the FDA’s principal deputy commissioner proclaimed during her keynote at last week’s NORD’s Rare Disease & Orphan Products Breakthrough Summit.…

Poland Mirosław Zieliński, president of the Polish National Forum for the Therapy of rare diseases (ORPHAN), the representative voice for 35 Polish rare disease associations, discusses the implications of the introduction of a national rare disease plan and the need to improve rare disease diagnostics. Furthermore, he highlights the importance of…

Austria Rudolf Widmann, CEO and founder of AOP Orphan, the first rare disease-specific European company, discusses the overriding goal to ensure rare disease patients are matched with the correct treatments and the importance of open dialogue with specialists and patient groups. Furthermore, he highlights the need to differentiate health technology assessments…

Pharma Having successfully accomplished a four-year turnaround exercise and with a fresh capital injection from a leading Italian PE firm, SIFI executive chairman Fabrizio Chines has ambitious growth objectives for the coming years, and is investing in R&D projects including the development of an ophthalmic orphan drug. The Italian pharmaceutical industry…

Pharma Ad Schuurman of the National Health Care Institute of the Netherlands (“Zorginstituut”) explains the main missions and mandates of this organization with regards to the basic insurance package covering all Dutch citizens, as well as its perception of pay-for-performance models, pan-European joint-reimbursement initiatives, and Zorginstituut’s policies on orphan drugs reimbursement and quality…

Pharma Prof. Luigi Naldini, Director of TIGET, discusses the success of the institute and the groundbreaking partnerships it has formed with the pharmaceutical industry, as well as his ambitions for TIGET over the coming years. Prof. Naldini, the San Raffaele Telethon Institute for Gene Therapy was set up in 1995 as…

Pharma The CEO of Onxeo, recently formed through the merger of the French BioAlliance Pharma and the Danish Topotarget, discusses its strategic turning decision to focus on orphan oncology, its promising pipeline of innovative products, her “positively opportunistic” attitude towards partnerships and international expansion, and the company’s overall strategy for differentiating…

orphan drugs Aegerion’s representative in Argentina discusses her efforts to raise awareness of Homozygous Familial Hypercholesteremia (HoFH), the rare disease targeted by her company’s ultra-orphan drug, and the remaining challenges of diagnosing patients. What is the current state of affairs for HoFH patients in Argentina? So far, as lomitapide is not yet…

orphan drugs Gabriela Pittis discusses the Argentinian healthcare system’s ability to diagnose, treat, and regulate rare diseases, and the country’s progress in further developing what is already the region’s most comprehensive system in this regard.   Is the Argentinian system able to reliably diagnose and treat rare diseases? “Rare diseases” is a…

Government Regulation As the only country in the G8 without an orphan drugs act, the President of Médunik, a company focused on bringing orphan drugs into the country, talks about the desperate need for better legislation in this area for the benefit of patients, and the misconceptions of treating Canada as a…