FDA Puts Blood Cancer Nods Under Scrutiny On Substantial Toxicity Profile Of PI3K Drugs

  • The FDA aims to put the PI3K inhibitor drug class under a more stringent regulatory scope as the inhibitor has demonstrated substantial toxicity.
  • In nearly all of the randomized clinical trials, patients randomized to the PI3K inhibitor arm experienced increased rates of fatal adverse events (AEs), serious adverse events (SAEs), Grade 3 or greater AEs, and treatment modifications or discontinuation due to AEs. 
  • Related: Gilead Withdraws Use Of Zydelig In Two Forms Of Blood Cancer In US.
  • Six randomized trials evaluating a PI3K inhibitor have demonstrated a higher rate of death or concerning OS results suggesting potential harm to patients, the FDA said in a briefing document ahead of an advisory committee meeting scheduled for Thursday.
  • Related: Incyte Pulls Parsaclisib US Application For Lymphoma Treatment.
  • The "unprecedented" observation raises a red flag for PI3K inhibitor approvals based on single-arm trials. 
  • The drug reviewers cannot correctly evaluate overall survival, the FDA says. Therefore, the agency says future PI3K approval should use randomized clinical trials with active comparators.
  • Read Next: TG Therapeutics Retracts FDA Request For Ublituximab / Ukoniq Combo In Blood Cancers.
  • The FDA also mentioned that drug development programs focused on determining the maximum tolerated dose in its review document.
  • Hence, lower doses, which may offer significant efficacy with reduced toxicity, are generally not explored extensively.
  • The FDA has approved four PI3K inhibitors for various blood cancers: Gilead Sciences Inc's GILD Zydelig (idelalisib), Bayer AG's BAYRY Aliqopa (Copanlisib), Secura Bio's Copiktra (duvelisib) and TG Therapeutics Inc's TGTXUkoniq (umbralisib). 
  • In 2020, Verastem Oncology VSTM divested Copiktra rights to Secura Bio for up to $381 million.
  • Novartis AG's NVS Piqray, a PI3K alpha-specific inhibitor approved for breast cancer, is not part of the group under FDA scrutiny.
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