Transplant surgery comes with various risks, one of which is infection that occurs when the patient’s immune system is weakened.Takeda’s drug is now the first FDA approved treatment For certain types of post-transplant infections that can be fatal.
The risk of infection comes from cytomegalovirus (CMV). Antiviral drugs can be used to treat CMV infection, but in some cases, the virus does not respond to treatment or develops resistance. Takeda’s drug maribavir was developed to treat CMV infections that do not respond to available antiviral drugs. This approval applies to 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 herpes virus. It is very common in humans, but usually does not cause any problems for people with healthy immune systems. In stem cell and organ transplant surgery, immunosuppressant drugs are a standard step to prevent the body from attacking the transplanted stem cells or organs. But the resulting weakened immune system also makes transplant patients more susceptible to infection. CMV infection can lead to the loss of transplanted organs or the failure of transplanted stem cells. In severe cases, CMV infection can lead to death.
Livtencity is a twice-daily oral drug designed to prevent the activity of the human cytomegalovirus enzyme pUL97, which is the key to all stages of viral infection. This method prevents the virus from replicating. Takeda’s drug application is based on the results of an open-label Phase 3 study that recruited 352 transplant patients who had CMV infection and responded to treatment. These patients were randomly assigned to receive Takeda drugs or one or two of the four antiviral drugs currently used to treat CMV. Treatment lasts up to eight weeks; then the patient is followed up for 12 weeks.
The purpose of the study was to compare the concentration levels of viral DNA in the blood at the end of the eighth week. In order to show efficacy, the virus concentration must be below a measurable level. According to FDA data, 56% of the 235 patients treated with Takeda’s drug reached this benchmark, and among the 117 patients who received treatment assigned by clinicians, this proportion was 24%.
The side effects reported in the study include taste disorders, nausea, diarrhea, vomiting and fatigue. The FDA stated that Livtencity may reduce the activity of two other antiviral drugs, ganciclovir and valganciclovir. Therefore, the agency recommends not to administer Takeda drugs together with these antiviral drugs. The FDA warned that virological failure, that is, the inability of the drug to suppress viral load, may be due to resistance to the therapy. The agency stated that CMV DNA levels should be monitored to check for this resistance.
Takeda said that Livtencity will be launched in the next few days.
“The transplant recipients have a long and complicated healthcare journey; Ramona Sequeira, President of Takeda’s U.S. Business Unit and Global Portfolio Commercialization, said in a statement: “With this With the approval of the therapy, we are proud to provide these people with a new oral antiviral drug to combat CMV infection and disease. ” Prepared statement.
FDA approves Aadi Bio’s drug to treat rare types of soft tissue cancer
Aadi Biosciences has a strong point in the new transformation of a decades-old drug Regulatory green light As a treatment for rare cancers, it became the first product approved by the FDA for a biotechnology company.
Aadi drug Fyarro is developed for treatment Perivascular epithelioid cell tumor (PEComas). These tumors form in soft tissues such as the stomach, intestines, lungs, female reproductive organs, and genitals, and are usually benign. Aadi, headquartered in Pacific Palisades, California, has developed drugs for the treatment of rare cases of PEComa tumor malignancy.
The FDA’s approval covers the treatment of PEComas that are already advanced and cannot be removed by surgery or cases where these tumors have spread. Regulatory decisions make Fyarro the first and only approved treatment for advanced malignant PEComa in adults.
Aadi’s intravenous drug is based on a decades-old immunosuppressant called sirolimus. This medication, also called rapamycin, is used to prevent organ transplant rejection. It works by blocking signal transduction in a pathway called mTOR. In addition to its role in the immune response, this pathway can also promote tumor growth. Aadi aims to develop Fyarro as a treatment method for cancer characterized by genetic changes in the mTOR pathway.
The new change that Aadi brings to sirolimus is to bind the molecule to albumin, a liver protein. According to the company, because albumin accumulates in tumors, combining the protein with drugs is a way to preferentially deliver it to tumors. If this method sounds familiar, it may be because it is similar to the method used by Abraxis Bioscience for its drug Abraxane, in which particles of chemotherapy paclitaxel are bound to albumin.
The FDA’s Fyarro decision was based on the results of an open-label Phase 2 study that recruited 34 patients. Three of the patients discontinued treatment due to adverse reactions. Of the remaining 31 patients in the study, 12 or 39% showed an overall response to treatment. Two of the patients achieved complete remission after a longer follow-up period. Among those who responded to treatment, 92% had a response that lasted 6 months or more, while 67% of responders had a response that lasted 12 months or more. In the two years after treatment, 58% of people had a response that lasted this long or longer.
Similar to other drugs in the mTOR class, Fyarro’s drug label warns that treatment may cause side effects, including oral swelling and ulcers, suppression of bone marrow activity, infection, and lower than normal potassium levels.
Aadi is relative to Nasdaq; it passed in August Reverse merger Cooperation with Aeropio Pharmaceutical Company. At the time, Aadi raised $155 million in funding from the investment company that bought the company’s shares. According to Aadi’s latest financial report, its cash position as of September 30 was approximately US$161.4 million.
Image courtesy of the Centers for Disease Control and Prevention
