March 2021

NNPDF Community,

I appreciate the opportunity to bring you the Spring edition of the NNPDF Board Chair update. As the world looks to rebound from the bitter year that was 2020, the optimism generated by the increasing availability of successful vaccines brings hope of better days ahead. While we are regularly reminded that we aren’t out of the woods yet, it’s encouraging to have hope. Thank you for doing your part in working toward a better tomorrow. 

The Niemann-Pick community has been putting in the work for a better tomorrow for over 30 years, and that work has brought us to a time when we have many potential therapies being studied. In our search for successful treatments for Niemann-Pick disease, there is a lot of news to share.

• We are fortunate to have two industry partners that are close to submitting their completed applications to the FDA for approval. Orphazyme has announced a targeted action/PDUFA date of June 17 for the FDA to consider arimoclomol for approval. Sanofi Genzyme will submit their application for olipudase alpha with the FDA later this year. We are hoping to soon become part of the 5% of rare diseases with an FDA-approved treatment.
• The pipeline for therapies in NPC shows great promise beyond arimoclomol. Intrabio and Cyclo Therapeutics have both announced positive results with their most recent clinical trials. Cyclo Therapeutics announced that they will begin enrolling for their pivotal trial soon while IntraBio is meeting with regulatory bodies around the globe to direct the next steps of their program in NP-C.
• Mallinckrodt’s announcement of the suspension of their clinical program has upended the lives of many in our community. The stories of clinical stability or improvement on this therapy that many of you have shared have reinforced our efforts to find a path forward for this therapy. We are partnering with leaders in our community in our ongoing efforts to advocate for patient access to this compound while exploring options for a path forward.

In our role as the national patient advocacy organization, the NNPDF is uniquely positioned to impact drug development through amplifying your voice in discussions with regulators, industry partners and payors. Joslyn’s background in market access is particularly important as we prepare for the approval of therapies and look to provide access to approved therapies for all Niemann-Pick patients. The health insurance literacy and access survey that our community completed has already proven to be an important tool for us in preparing our community for FDA-approved therapies.

Reflecting back on our community’s experience with clinical trials, we have come to know the difficulties of conducting traditional randomized clinical trials in an ultra-rare disease. Wide spectrums of disease with variable progression, limited numbers of patients, and extended use of placebo all hinder our ability to conduct successful traditional placebo-controlled trials with Niemann-Pick patients.    

Right now, it is critical that regulators understand the needs of our community. As such, NNPDF has scheduled a Listening Session with the Center for Drug Evaluation and Research (CDER), a branch of the FDA. During this session, we will call attention to the unmet need in both the ASMD and NPC spaces. Even with the hopeful approval of olipudase, there will be unmet need for the neurologic sequala of ASMD. We will also highlight the ongoing difficulties with designing and conducting traditional clinical trials in our patient community and urge the FDA to allow for unique clinical trial designs that assess endpoints that matter to Niemann-Pick patients and families. We will advocate for a collaborative approach to these issues because we need the patient voice to not only be heard, but to be part of the regulatory process. We will provide follow up to that meeting in the next newsletter.

In our last board meeting, we discussed the importance of remaining connected as a community. For thirty years, the NNPDF has been the vehicle that allowed families to connect with one another and support each other. While last year’s virtual conference allowed a record number of attendees to connect with each other, the void of in-person connections has left many of us feeling less connected with our Niemann-Pick community. We are so fortunate to have Laurie Turner leading our Family Services and hosting the many virtual connection opportunities. The foundation will continue to expand our virtual reach through many different platforms including the launch of a new website this summer. We urge all of you to stay connected with each other and support each other because we need each other now more than ever. We are all looking forward to the day where we can see you all in person. Until then, be well and enjoy your Spring.

Thank you all for your efforts in supporting each other, our community and the NNPDF. Please reach out to me or any of your board members with any questions, concerns or ideas. 

Best wishes,

Justin Hopkin, MD
NNPDF Board Chair
[email protected]

Supporting One Another. Supporting Our Community.