Community Update on ISIS-SMNRx from Biogen and Isis

Biogen and Isis Pharmaceuticals recently provided an update on the clinical development of ISIS-SMNRx, which is currently being tested in Phase 3 clinical trials:

Dear members of the SMA community,

Over the past few months, we have received many questions from members of the SMA community about ISIS-SMNRx and when it might be widely available to people with SMA. We are deeply moved by your stories and recognize the urgent need within the SMA community for patients and families living every day with this devastating disease. We wanted to provide you our thoughts on key issues related to the ISIS-SMNRx program. We understand that words alone do not give you the comfort and peace of mind you deserve, but we hope this letter will provide answers to some of your questions.

First, we want to assure you we are doing everything in our power to advance ISIS-SMNRX though development, and hopefully approval, as quickly as possible. While the drug development process can be frustratingly slow, especially to patients and families touched by a disease like SMA, the development program for ISIS-SMNRx has progressed much faster than most. An early and important step in moving this investigational drug forward was receiving Fast Track designation from the FDA. Fast Track is designed to facilitate the development and expedite review of investigational drugs for approval to treat serious conditions such as SMA. This designation allows us more frequent meetings with the FDA throughout our clinical program to help us gain insight and guidance from the agency as we work toward a regulatory filing. Our hope is these more frequent discussions offer the type of guidance that will result in fewer delays and quicker reviews. The communications with regulators has been ongoing and we continue to engage in regular and productive discussions with health authorities in the US and across the EU to understand the most expeditious path forward for ISIS-SMNRx.

In addition to Fast Track designation, there are several additional regulatory approaches in the U.S. that are designed to expedite the investigational drug review process. These include Accelerated Approval, Breakthrough Therapy, and Priority Review. We have and will continue to explore all of these approaches and pursue every option that may accelerate the possible approval of ISIS-SMNRX. However, we have to continue the well-controlled Phase 3 clinical trials to collect the data necessary for regulators to make decisions about the safety and efficacy of ISIS-SMNRx.

A question many parents and patients with SMA ask is how to interpret the open-label, Phase 2 clinical trial results to date. Like all of you, we are encouraged by the results. However, the current open-label data, while exciting and supportive of continued research, are not from definitive randomized well-controlled trials. We remain committed to executing the Phase 3 pivotal studies as quickly as possible to build the body of evidence needed for evaluation by regulators in the U.S. and around the world.

We are driven in our work on ISIS-SMNRx by the acute awareness that patients and families are waiting for access to treatment. Our current focus is to advance our ongoing well-controlled Phase 3 studies which will be required for approval. We believe that this approach is the best path to making this investigational drug available to the SMA community in the shortest period of time for the greatest number of patients. Patients and families frequently ask us about access to this investigational drug prior to regulatory approval and the timeline associated with opening an Expanded Access Program (EAP). This is among the most difficult questions we receive and unfortunately, there are easy no answers.

Our goal is to hopefully make ISIS-SMNRx available to patients once we are confident that doing so will not risk slowing the collection of the data necessary for regulators to make a determination about the safety and efficacy of ISIS-SMNRx and once we are able to end the well-controlled study arm. Making ISIS-SMNRx available earlier in the process could jeopardize potential approval and ultimately access for patients around the world. While we would like to provide more details about the timing of an Expanded Access Program for ISIS-SMNRx, these variables currently prohibit us from offering a specific timeline. We will continue to work diligently with regulators and communicate more information to all of you as soon as we can.

Patient and community engagement is vital to the success of any clinical program, especially in rare diseases such as SMA. We want to thank you for the important contributions this community has already made in building disease awareness and sharing patient and caregiver experiences with us and regulatory agencies. For example, your poignant and powerful “SMA Voices” publication will play a vital role in helping regulators understand community needs and the impact of SMA on patients and families.

We continue to welcome dialogue with the entire SMA community. We want you to know that while there are times when we may need to remain silent, we are committed to providing as much information as we can share as soon as we are able to share it. We remain steadfast in our collective goal to advance this program in order to hopefully have this investigational drug approved and available to as many patients as possible as quickly as possible.

Biogen and Isis Pharmaceuticals

Additional Background Information

Download a copy of Learning about Clinical Trials

Learn about our “Voices of SMA” project

Read an update on Cure SMA’s FDA Engagement Initiative

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