August 28, 2018
BIOINVEST BREAKING NEWS: Ionis (IONS)
IONS/AKCA Receive CRL From FDA for Waylivra Despite Positive AdCom
In a somewhat surprising move, ONS/AKCA received a complete response letter (CRL) for Waylivra (volanesorsen) from the FDA. It is also unexpected given the 12-8 Advisory Committee vote in favor of approval on May 10th (https://www.biospace.com/article/fda-advisory-committee-gives-green-light-to-akcea-therapeutics-rare-disease-drug-waylivra/) and the agency’s recent accommodative stance towards new drugs for rare diseases such as IONS’ approval of Spinraza. We believe the FDA focused on the thrombocytopenia seen in registration trials. However, we note that IONS has taken steps to prevent these (all treatable) bleeds since those studies were finished. Furthermore, FCS patients from Waylivra’s two pivotal studies have been allowed to continue receiving the treatment in an extension phase. The companies plan to request an FDA meeting as soon as possible to discuss the next steps and they believe additional data from ongoing studies could potentially be helpful in addressing the agency’s concerns. Our expectation is roughly a delay of one-two months until further clarity is delivered after IONS/AKCA meet with the agency.
In our view, there is no a direct read-through from the Waylivera CRL on the decision pending Tegsedi (6-Oct PDUFA). Part of our confidence for a FDA approval is based on the fact that Tegsedi is already approved in the EU. Also supportive of a Tegsedi approval is the pipeline safety profile where zero cases of thrombocytopenia having been reported across IONS’ 13 LICA programs. While we are disappointed in the Waylivra CRL, the drug still has a potential path to the market. Importantly, in our view the Waylivra safety concerns have no read through to the rest of the IONS/AKCA pipeline