Tagged with Rare Diseases

Switzerland Dipharma SA is a Swiss specialty pharmaceutical company, developing improved generics medicines for rare metabolic diseases. Founder & CEO Marc-Olivier Geinoz discusses why the company has chosen to compete in the largely unexplored rare diseases generics space, its direct presence in the German market, and the opportunities inherent in metabolic…

USA With Rare Disease Day coming up on February 28th, one of the leading organizations dedicated to serving affected patients in the US, the National Organization for Rare Disorders (NORD), is making a push to put the spotlight on the limited access to diagnosis, treatment and care suffered by rare disease…

USA Following on from our profile on five of the most promising clinical-stage biotech companies developing orphan therapies in Europe, here we move to the world’s biggest biopharmaceutical market and R&D hub, shining the spotlight on five of the most important and innovative rare-disease start-ups in the USA.   The US…

Europe With Rare Disease Day falling on 28 February, PharmaBoardroom dedicates this month to exploring the global rare disease landscape, highlighting key trends and developments as well as profiling the most innovative global players in this space. We start the party off with five of the most promising clinical-stage biotech companies…

Switzerland A trained physician with several years of practice under her belt, Katharina Gasser then moved into clinical research as an investigator before joining the pharma industry in 2005. Now head of Biogen’s Swiss affiliate and chair of the executive committee of industry association Interpharma, Gasser cites her diverse background as…

Global The Top 10 rare disease companies globally, ranked by worldwide orphan drug sales for 2018. Celgene takes top spot, followed by Roche and Novartis. Made with Visme Infographic Maker

Global In a wide-ranging and exclusive interview with PharmaBoardroom, Ipsen CEO David Loew looks back on an atypical first 100 days at the helm of the iconic French mid-cap; its EUR 3 billion war chest for bolt-on acquisitions; planned expansions in rare- and ultra-rare diseases; and the niche oncology segments where…

USA Rare disease – defined in the US as a disease with a maximum patient population of 200,000 – is perhaps the most salient issue at the nexus of innovation, access, and affordability in the country.   As FDA CDER director Dr Janet Woodcock indicates, “rare diseases have been rising as…

Switzerland Pierrre Morneau of Takeda Switzerland explains the therapeutic areas at the core of the company, equitable access, the Swiss framework for rare diseases, and the major challenges he is dealing with today.   The environment is shifting at a more rapid speed than ever before. Past paradigms are obsolete Having…

USA Zogenix’s Dr Stephen Farr shares his remarkable career journey from academia to industry, the company’s transition from pain therapeutics into rare diseases, product launch strategies, access and affordability issues, and talent attraction challenges.   While it is true you can make great contributions in academia, I think you can make…

USA Takeda USA’s Ramona Sequeira outlines her expanded role as president of global portfolio commercialization, the crucial importance of investing in culture, and the company’s multifaceted response to the COVID-19 crisis.   In general, through the global COVID response, we’ve seen the walls between companies and across sectors come down as…

USA Brad Campbell, president and COO of US rare disease specialist Amicus, talks domestic and global product launches, the company’s first steps into the gene therapy space, and how a biotech success story like Amicus can be a guiding light in market access and pricing discussions. Ultimately, we envision our competition…