FDA grants approvals for transplant drug Takeda and cancer therapy Aadi Bio


Transplant procedures carry all kinds of risks, including infections that develop in a patient’s weakened immune state. A drug Takeda Pharmaceutical is now the first FDA-approved treatment for a particular type of post-transplant infection that can become fatal.

The risk of infection comes from the cytomegalovirus (CMV). Antivirals are available to treat CMV infections, but in some cases the virus does not respond to treatment or develops resistance. Takeda’s drug, maribavir, was developed to treat CMV infection that does not respond to available antivirals. The approval covers patients 12 years of age and older who weigh at least 35 kg (approximately 77 pounds). Takeda will market its new drug under the name “Livtencity”.

CMV is a type of herpes virus. It is commonly found in humans, but is generally not a problem in those with healthy immune systems. In stem cell and organ transplant procedures, immunosuppressive drugs are a standard step to prevent the body from attacking the transplanted stem cells or organ. But the resulting weakened immune system also makes transplant patients more susceptible to infections. CMV infection can lead to the loss of a transplanted organ or the failure of the transplanted stem cell transplant. In severe cases, CMV infection can lead to death.

Livtencity, a twice-daily oral medication, is designed to inhibit the activity of the human cytomegalovirus enzyme pUL97, an enzyme that is essential at various stages of viral infection. This approach blocks viral replication. Takeda’s drug application was based on the results of an open-label Phase 3 study involving 352 transplant patients who had CMV infections who responded to treatment. These patients were randomly assigned to receive either the drug Takeda or one or two of the four antivirals currently used to treat CMV. Treatment was continued for up to eight weeks; the patients were then observed for a 12 week follow-up period.

The aim of the study was to compare the levels of DNA concentration of the virus in the blood at the end of the eighth week. To show efficacy, the viral concentration had to be below measurable levels. According to the FDA, of the 235 patients who received the drug from Takeda, 56% of them reached this benchmark compared to 24% of the 117 patients who received clinician-assigned treatment.

Side effects reported in the study included taste disturbance, nausea, diarrhea, vomiting, and fatigue. The FDA has said Livtencity may reduce the activity of two other antiviral drugs, ganciclovir and valganciclovir. Therefore, the agency recommends that the drug Takeda not be administered alongside these antivirals. The FDA has warned that virologic failure, in which a drug fails to suppress the viral load, can occur due to resistance to therapy. The agency said CMV DNA levels should be monitored to check for such resistance.

Takeda said Livtencity will be available in the coming days.

“People who have a transplant have a long and complex healthcare journey; with the approval of this treatment, we are proud to offer these individuals a new oral antiviral to fight CMV infection and disease, ”said Ramona Sequeira, president of the US business unit of Takeda and the marketing of the global portfolio, in a press release.

FDA Approves Aadi Bio’s Drug for Rare Type of Soft Tissue Cancer

Aadi Biosciences’ new version of a decades-old drug has the regulatory green light for the treatment of a rare form of cancer, making it the first biotech product approved by the FDA.

Aadi’s drug, Fyarro, was developed as a treatment for perivascular epithelioid cell tumors (PEComas). These tumors, which form in soft tissues such as those in the stomach, intestines, lungs, female reproductive organs, and genitals, are usually benign. Pacific Palisades, Calif., Aadi developed his drug for the rare cases where PEComa tumors become malignant.

The FDA approval covers the treatment of PEComas that have advanced and cannot be removed surgically, or cases in which these tumors have spread. The regulatory decision makes Fyarro the first and only approved treatment for advanced malignant PEComa tumors in adults.

Aadi’s intravenous drug is based on a decades-old immunosuppressant called sirolimus. This medicine, also known as rapamycin, is used to prevent organ transplant rejection. It works by blocking signaling on a pathway called mTOR. In addition to its role in the immune response, this pathway can also promote tumor growth. Aadi aimed to develop Fyarro as a treatment for cancers characterized by alterations in the genes of the mTOR pathway.

Aadi’s new twist on sirolimus binds the molecule to albumin, a protein in the liver. According to the company, since albumin accumulates in tumors, binding of this protein to the drug allows it to be delivered in preference to tumors. If the approach sounds familiar to you, it may be because it is similar to the approach used by Abraxis Bioscience for its drug Abraxane, in which the particles of the chemotherapy paclitaxel are bound to albumin.

The FDA’s Fyarro decision was based on the results of an open label Phase 2 study involving 34 patients. Three of these patients discontinued treatment due to side effects. Of the remaining 31 patients in the study, 12, or 39%, showed an overall response to treatment. Two of these patients achieved a complete response after a longer follow-up period. Of those who responded to treatment, 92% had a response lasting six months or more, while 67% of responders had a response lasting 12 months or more. After two years after treatment, 58% had a response of as long or longer duration.

Similar to other drugs in the mTOR class, the Fyarro drug label warns that treatment may cause side effects, including swelling and sores in the mouth, suppression of bone marrow activity, infections and lower than normal potassium levels.

Aadi is relatively to the Nasdaq; it entered the public markets in August via a reverse merger with Aerpio Pharmaceuticals. At that time, Aadi raised $ 155 million in funding from investment firms who bought shares of the company. According to Aadi’s most recent financial report, his cash position as of September 30 was approximately $ 161.4 million.

Image from the Centers for Disease Control and Prevention


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