Tagged with Rare Disease

USA William H Lewis, chairman and CEO of serious and rare disease specialist Insmed, talks through the impact of recent product approvals in the US, preparations underway for international launches, the impact of COVID-19 on Insmed’s operations, and the firm’s highly promising clinical pipeline.   The most important element of any…

Global Speaking exclusively to PharmaBoardroom, Sanofi Genzyme Executive VP Bill Sibold outlines Sanofi’s strategy for its specialty care unit, the wealth of potential breakthrough assets in its pipeline, product launch strategies, and the post-COVID industry of tomorrow.   Since I joined in 2011, it feels as if my time with Sanofi…

USA French firm LFB is targeting the lucrative US market with its hemophilia products and recently received two FDA approvals. Although launching new products in the midst of a global pandemic is not without challenges, LFB USA CEO Jose Antonio Moreno Toscano is optimistic that the launches will be a success…

Korea After failing to attain reimbursement for its Amyotrophic Lateral Sclerosis (ALS) treatment NEURONATA-R® in Korea, Corestem has its sights set on the US market and an Investigational New Drug (IND) approval from the FDA.   Dr Kyung Suk Kim, Corestem’s CEO, recently spoke to PharmaBoardroom to give an update on…

Korea Kyung Suk Kim, CEO of Corestem, the first company to receive approval for a stem cell therapy for Amyotrophic Lateral Sclerosis (ALS) in Korea, recounts the firm’s main achievements since her previous interview with PharmaBoardroom in 2018. Dr Kim unveils Corestem’s new strategy to prioritise the US market and offers…

USA As of 11 June 2020, there have been over two million confirmed cases of COVID-19 in the US and over 115,000 deaths, with the US having occupied the leading spot globally in terms of confirmed cases since late-March. While the pandemic has wreaked havoc across the entire country, advocates of…

USA Peter L Saltonstall, president and CEO of the US National Organization for Rare Disorders (NORD) shares the organization’s key priorities, what makes NORD a unique patient advocacy group in the US, prevailing access and affordability issues for American rare disease patients, and how the country should continue to incentivize a…

Belgium Geert Van Hoof, founding general manager of Chiesi Belgium, shares the exciting milestones of the company’s first decade in Belgium, including their stunning year-on-year double-digit growth, their market leadership in respiratory as well as neonatology, and their growing presence in rare diseases, as well as the attractiveness of the Chiesi…

Czech Republic A cross-industry push for better and broader access to innovative treatments is underway in the Czech Republic. Ripe for reform is the country’s orphan drug legislation as no standard pathway for their assessment, pricing and reimbursement currently exists.   [For cancer treatment the country has been] quite successful in moving…

Mexico Chiesi Mexico’s Marco Ruggiero discusses his initial impressions of the Mexican pharma market, his strategic priorities for the affiliate, and the increasing importance of the company’s respiratory portfolio in Mexico.   With respiratory diseases, there is sometimes the erroneous perception that they are not as severe or urgent as other…

Mexico David López García, Mexico country manager of global rare genetic therapy player Biomarin, outlines the challenging situation around patient access to orphan drugs in Mexico and the importance of building awareness of rare diseases.   The challenge for orphan drugs is the price. It is not always easy to explain…

Italy Italian mid-cap firm Chiesi is continuing its expansion in the USA market with the launch of a Boston-based subsidiary focusing on rare and ultra-rare diseases.   We are very excited to put Chiesi’s decades of experience in drug development and dedication to patients to work to make a positive difference…