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Sanofi's head of rare diseases discusses hemophilia acquisition, research

? The pipeline in rare diseases is clustered around four therapeutic areas: rare nephrology, rare hematology, a rare neurology and neuromuscular area, and rare pediatric and metabolic.

? Following the Bioverativ acquisition, rare hematology will be the largest rare disease area.

? Sanofi intends to bring "a broad tool kit" of medicines to address hemophilia.

Rand Sutherland, head of rare disease research at Sanofi, spoke to S&P Global Market Intelligence about how the recent acquisition of rare disease company Bioverativ Inc. and the restructured deal with Alnylam Pharmaceuticals Inc. on fitusiran for hemophilia will knit together at the Paris-based pharmaceutical group.

The conversation has been edited and condensed for brevity and clarity.

SNL Image

Rand Sutherland
Source: Sanofi

S&P Global Market Intelligence: How does the acquisition of Bioverativ fit in with the recent restructuring of hemophilia treatment fitusiran with Alnylam?

Rand Sutherland: From an R&D perspective, we see this as an area in which there's value in consolidation, potentially of multiple assets and projects. Now, our transaction is tentative at this time, so I am speaking theoretically. But we see the replacement factors as a continuing area of significant focus. Bioverativ has significant presence in that market and has an R&D pipeline that continues to focus on what improvements can be made in extending the half-life of these factors.

And there has been tremendous novel innovation emerging over the past month and years in this area, with gene therapy and other innovations including Hemlibra and fitusiran. We really see this as an area that we can most effectively enter by having a broad tool kit and working, over time, to really help identify which patients are most appropriate for, and would benefit most from, these various medicines.

So that's our approach: To really try to bring a complementary and full suite of medicines to patients with hemophilia, and do so in the most scientific and informed way possible.

With the recent approval of Roche Holding AG's Hemlibra, hemophilia is getting a lot of attention. Is there a danger that there will be too many competing products in this marketplace?

Our view is that progress is good for patients. We anticipate that the established medicines, the replacement factors, will continue for the foreseeable future, to be a mainstay of therapy. And so a relationship with Bioverativ and having those marketed products as part of our commercial portfolio will allow us to come in and be major players in that space. Because of the established efficacy and the long history there, there's a possibility that there will naturally be some barriers to uptake and those barriers to uptake are going to be overcome by data and by clinical experience.

Our view is that there will be room for established innovation and there will also be room for evolution based on the data that is generated over time, and we see that opportunity to generate the new data as really a way to contribute to the overall care of patients with hemophilia. So I think we see it more as an opportunity than a risk, recognizing that the data will emerge and strategic decisions that we make over time will inform how that landscape evolves.

How did you develop your interest in rare diseases?

I am a pulmonologist by background. I was in academia actually until about four years ago, as a professor of medicine at the University of Colorado and worked at an institute there in Denver focused on rare and difficult to treat pulmonary and immunological disorders. My academic focus in the research side was on severe asthma.

Clinically I was responsible for a group that took care of a lot of different rare pulmonary diseases including cystic fibrosis, pulmonary fibrosis and others. And so it's from that perspective that I've now moved from pulmonary into rare diseases, which really require a broad therapeutic view; rare diseases are defined by frequently a genetic basis rather than by any specific organ system, and so diversity of clinical experience and background I think is useful in this regard.

Will you use your platform to expand into other areas of rare disease?

I would say that one area where we had some expansion over the course of the last year is in the rare nephrology area. Then we also have our Parkinson's disease program, which sits in the neurology group also with some projects in rare eye disorders.

In terms of the bigger picture in pharmaceutical research, how can we explain the intense interest in rare diseases while at the same time research into serious diseases such as Alzheimer's disease, which affect so much more of the population, is being discontinued by some significant players?

So those are macro trends in the industry that I can't really explain so I am just commenting here as an individual. The hope is that as an industry we continue to innovate both in rare diseases and in more prevalent diseases there's tremendous unmet need. The decisions that individual companies make are likely based on a combination of factors including strategic focus, success rates, the need to prioritize and even for big companies like ours, it's hard to do everything well.

And so there's a balance of everybody doing everything at the same time, versus prioritizing and focusing, where you have your strengths, where you have experience, legacy and I think in our case, in rare diseases, where you have a longstanding presence in research, development and commercialization, treatment of patients, interaction with patients, patient families, patient association, and global leaders and I think that we do our work best when we work in that way.

And we have the advantage of being able to leverage all of that experience and history and so that's one of the reasons for our significant focus on rare diseases. So without commenting on the strategy of others, I think what works well for us is really doubling down, being focused and attentive to what it is that we think we can do well and that's what's behind how we think about our strategy in rare disease here.