EMA grants Conditional Approval for Ataluren
Ataluren, PTC Therapeutics’ nonsense suppression drug received Conditional Approval. This is a big moment for Duchenne, a landmark decision and a sign of hope for all. While Ataluren is targeted at a small subset of patients (13% +/-), the conditional approval is a sign that the EMA worked very hard to understand Duchenne, that they recognize this unmet need, the progressive and debilitating process of Duchenne and the urgent need to treat.
I recognize it is a bittersweet moment for every family whose son, grandson, cousin, friend does not fit the criteria for Ataluren, as every person diagnosed needs and deserves access to treatments. The amazing and wonderful news is that once one drug is approved, there is a path forward for all, a door has opened, a regulatory agency intimately familiar with weighing benefit and risk, safety and efficacy, a regulatory agency that recognizes need and urgency, and agrees to move forward.
But the first approval is, well a FIRST, a sign of change, a gateway to understanding and recognition that regulatory agencies understand that rare disease must necessarily be viewed through a different lens than common disease, that the patient voice and their view of benefit and risk is integral to the process, that the progression of the disease is well known and from the day DUCHENNE is diagnosed, there is little uncertainty about progression. It is well known and is on replay in the minds and hearts of everyone who has ever known and loved an individual with the diagnosis.
Conditional approval is used by the EMA. It enables individuals who fit the criteria to access the compound and ensures reimbursement. It also requires that the sponsor (PTC Therapeutics) complete the Phase III study, submit the full application to the EMA for analysis and a final decision for full approval.
Conditional or Adaptive licensing is not currently used in the US. In our white paper (Putting Patient’s First…) we recommended adaptive licensing/conditional approval, suggesting that once safety is established and a trend toward benefit, conditional approval is an appropriate vehicle, recognizes the urgent needs and provides access.
While the label for the Conditional Approval (5+ ambulatory) is narrow, PTC plans to conduct studies in young children, as well as, the non-ambulant population. PTC recognizes that ALL individuals with a nonsense mutation need and deserve access to therapies and is working with UPPMD to organize a webinar with the next 10 days. They will describe their plans for moving forward with the FDA and answer questions you may have.
Take just one moment and celebrate today. This is a very good day for all of us, but our work is not complete. We have a pipeline full of hope and need to continue to expand our robust pipeline. The MD-CARE ACT has played a major role and needs to continue to serve this community. The Draft Guidance is nearing completion and will be submitted to the FDA. Members of the Steering Committee and Working Groups have suggested the Guidance should be submitted to EMA, as well, in an effort to promote harmonization in the US and Europe. And together we have worked to raise awareness, encourage regulatory agencies to understand this urgent need.
And today, the EMA’s decision is a good sign – Strength Happens Together.