A Focus on Both Scientific Development, Sales

Q: The annual 2018 BioMarin report mentions an increase in demand for Naglazyme in Latin America and Europe. What is the demand in Mexico?

A: There is a high prevalence of the Mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy Syndrome) in Latin America since the region contains 400 of the 1,000 patients estimated to exist worldwide. However, compared to other countries in the region, Mexico has a lower demand for this orphan drug due to a lower prevalence. Another product called Vimizim has the potential to generate more demand in the Mexican market. Vimizim has the potential to treat around 100 people suffering from Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome) in the country and it has a wider distribution. This orphan drug is projected to have the highest demand in Mexico.

Q: Given the difficulty of commercializing orphan drugs in Mexico, how does BioMarin’s business model differ from others in the market?

A: A combination of knowledge and expertise is necessary to diagnose rare diseases and thus be able to prescribe an orphan drug. BioMarin’s business model is different from other biogenetic companies because we can focus on both scientific developments and sales. The remuneration we obtain from marketing our products allows us to boost investment in new therapies.

Q: What expectations does BioMarin have for COFEPRIS’ new Committee for Special Attention for Rare Diseases?

A: Regarding the creation of this new division, we really hope it will lead to a simplified registration process for our products; however, the main problem does not depend on COFEPRIS. BioMarin manufactures orphan drugs for rare diseases with a small patient base and it faces several access barriers in other steps of the process to market its products. There are three steps to follow: the registration with COFEPRIS, the clinical/HTA audit with the General Council of Health and the sale of our products to health institutions. Our problem arises in the third step because we must convince several payers such as IMSS, ISSTE, Seguro Popular and PEMEX to consider us in their budgets.

Q: What is your agenda with the Mexican Federation of Rare Diseases (FEMEXER) and with the Commission for the Analysis, Evaluation, Registration and Monitoring of Rare Diseases (CAERSER)?

A: The relationship BioMarin has with organizations like FEMEXFER is key to our development because we are a publicly-listed company and industry regulations do not allow us to have direct contact with patients. We also believe that the foundation of CAERSER was key for the industry because it is necessary to increase the visibility of rare diseases. We hope for a rapid development of the CAERSER projects and that these types of organizations will help to further open the government to these drugs because no new orphan drug has been introduced since 2011 at the major payers (IMSS/Seguro Popular).

Q: What concrete actions does BioMarin carry out to improve genetic research in Mexico?

A: Mexico is not yet an ideal country for research in gene therapy but the implementation of some improvements in the health and regulatory systems would create a better environment for companies like BioMarin to improve local investments. Our expectations for 2018 are to win the approval of Vizimim to help those who suffer from Morquio A Syndrome and to establish stronger alliances with the government to accelerate the release of our product.

Q: BioMarin will produce biologics after acquiring Pfizer’s biologics manufacturing plant in Ireland. What is the expected impact for Mexico?

A: The plant in Shanbally, Ireland, is an important asset in manufacturing for the commercial diversification of BioMarin’s production because it helps us not to be dependent on a single center. A big part of our production is in Shanbally and this fact guarantees the drug availability to the Latin American patients.