FDA Action Alert: Takeda, Aadi and Fennec


The U.S. Food and Drug Administration (FDA) has a few PDUFA dates for the Thanksgiving Holiday week. Here’s a look.

Takeda’s Maribavir for CMV Infections After Transplant

Takeda Pharmaceutical has a target action date of November 23, 2021, for its maribavir as treatment for post-transplant recipients with refractory cytomegalovirus (CMV) infection with or without resistance. The FDA’s Advisory Committee unanimously recommended its approval on October 7. The recommendations were based on data from the Phase II and III trials.

Maribavir is an orally bioavailable anti-CMV compound, an antiviral drug that targets and inhibits the UL97 protein kinase and its natural substrates. It has received Orphan Drug Designation by the European Commission and by the FDA. The FDA also granted it Breakthrough Therapy Designation.

“The treatment of CMV in patients who have undergone a solid organ or stem cell transplant is complicated, especially in patients who have failed standard treatment and who may be at risk for side effects from currently available medications,” said Emily Blumberg, Director, Transplant Infectious Diseases, Penn Medicine. “I am excited about the potential for an additional treatment option for post-transplant patients with CMV.”

Aadi Biosciences’ Fyarro for PEComa

Aadi Bioscience has a target action date of November 26 for its New Drug Application (NDA) for Fyarro (sirolimus albumin-bound nanoparticles for injectable suspension, nab-sirolimus ABI-009) for treatment of PEComa. The drug is an mTOR inhibitor bound to human albumin. PEComa is a rare, aggressive sarcoma. There are no approved therapies.

The NDA is built on data from the AMPECT trial which demonstrated meaningful clinical efficacy in malignant PEComa.

At the company’s third-quarter financial report on November 10, Neil Desai, founder, chief executive officer and president of Aadi said, “Following the successful merger completed in the third quarter, we are now fortified on all fronts with a strengthened balance sheet and expanded management team ahead of our November 26 Prescription Drug User Fee Action target date for Fyarro. With a sizeable PIPE financing that closed in connection with our merger with Aerpio, we can effectively deploy capital toward the commercialization of Fyarro in advanced malignant PEComa and for our planned tumor-agnostic registrational trial in patients with solid tumors harboring inactivating alterations of TSC1 and TSC2 genes in the mTOR pathway.”

Fennec’s Pedmark for Ototoxicity Induced by Chemotherapy in Children

Fennec Pharmaceuticals had a target action date of November 27 for the resubmission of its NDA for Pedmark (a unique formulation of sodium thiosulfate) for ototoxicity (hearing loss) induced by cisplatin chemotherapy in patients one month to less than eighteen years of age with localized, non-metastatic solid tumors. The drug was granted both Fast Track and Breakthrough Therapy Designations by the FDA. The company received a Complete Response Letter (CRL) on August 10, 2020, that referred to problems with the facility where the drug was manufactured, but no clinical safety or efficacy problems were noted and there was no requirement for additional clinical data.

Cisplatin and other platinum chemotherapies are used to treat many pediatric cancers. Unfortunately, these cause damage, typically permanent, to hearing. The drug has been studied by co-operative groups in two Phase III trials of survival and reduction of ototoxicity in childhood cancers usually treated with intensive cisplatin therapy, such as newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, medulloblastoma, and other solid tumors.

At the company’s third-quarter financial report on November 10, Rosty Raykov, chief executive officer of Fennec, said, “We continue to work with the FDA on their review of our NDA application, in advance of the pending Pedmark PDUFA target action date of November 27. We are focused on essential activities in preparation to bring this important treatment to children receiving cisplatin chemotherapy.”

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