FDA approves new treatment for refractory multiple myeloma

FDAToday, the U.S. Food and Drug Administration granted accelerated approval to Xpovio (selinexor) tablets in combination with the corticosteroid dexamethasone for the treatment of adult patients with relapsed refractory multiple myeloma (RRMM) who have received at least four prior therapies and whose disease is resistant to several other forms of treatment, including at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody.

"While there is no cure for multiple myeloma, there are FDA-approved treatments to target the cancer and slow down the spread of the disease. Sadly, often over time, patients can exhaust all available treatments and still see their disease progress," said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research. "Today we approved a treatment under our accelerated approval program that provides a treatment option for patients with multiple myeloma with no available therapy."

Multiple myeloma is cancer that begins in plasma cells (white blood cells that produce antibodies) and may also be referred to as plasma cell myeloma. Abnormal plasma cells build up in the bone marrow, forming tumors in many bones of the body. As more antibodies are made, it can cause blood to thicken and keep the bone marrow from making enough healthy blood cells. The exact causes of multiple myeloma are unknown, but it is more common in older people and African Americans.

Efficacy was evaluated in 83 patients with RRMM who were treated with Xpovio in combination with dexamethasone. At the end of the study, the overall response rate was measured at 25.3%. The median time to first response was four weeks, with a range of one to ten weeks. The median duration of response was 3.8 months. The efficacy evaluation was supported by additional information from an ongoing, randomized trial in patients with multiple myeloma.

Common side effects of patients taking Xpovio in combination with dexamethasone include a low white blood cell count (leukopenia), a low count of neutrophils, a type of white blood cell (neutropenia), low count of platelets (thrombocytopenia) and low amount of red blood cells (anemia). Patients also reported vomiting, nausea, fatigue, diarrhea, fever, decreased appetite and weight, constipation, upper respiratory tract infections and low blood sodium levels (hyponatremia).

Health care professionals are advised to monitor patients for low blood counts, platelets and sodium levels. Patients should avoid taking Xpovio with other medications that may cause dizziness or confusion and avoid situations where dizziness may be a problem. Health care professionals are advised to optimize the patient's hydration status, blood counts and other medications to avoid dizziness or confusion. The FDA advises health care professionals to tell females of reproductive age and males with a female partner of reproductive potential to use effective contraception during treatment with Xpovio. Women who are pregnant or breastfeeding should not take Xpovio because it may cause harm to a developing fetus or newborn baby. Xpovio must be dispensed with a patient Medication Guide that describes important information about the drug's uses and risks.

Xpovio in combination with dexamethasone was granted accelerated approval, which enables the FDA to approve drugs for serious conditions to fill an unmet medical need based on an endpoint that is reasonably likely to predict a clinical benefit to patients. Further clinical trials are required to verify and describe Xpovio's clinical benefit.

The FDA granted this application Fast Track designation. Xpovio also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The FDA granted the approval of Xpovio to Karyopharm Therapeutics.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Most Popular Now

Preventing tumor metastasis

Researchers at the Paul Scherrer Institute, together with colleagues from the pharmaceutical company F. Hoffmann-La Roche AG, have taken an important step towards the dev...

A new drug could revolutionize the treatment of ne…

The international team of scientists from Gero Discovery LLC, the Institute of Biomedical Research of Salamanca, and Nanosyn, Inc. has found a potential drug that may pre...

Interactions discovered in cells insulating nerve …

Schwann cells form a protective sheath around nerve fibres and ensure that nerve impulses are transmitted rapidly. If these cells are missing or damaged, severe neurologi...

Breast cancer can form 'sleeper cells' after drug …

Breast cancer medicines may force some cancer cells into 'sleeper mode', allowing them to potentially come back to life years after initial treatment. These are the early...

Anniversary of the pivotal RE-LY® trial marks a de…

Boehringer Ingelheim today announces the ten-year anniversary of the RE-LY® trial publication(1-3) recognising the contribution made in the decade since by patients, heal...

Experimental validation confirms the ability of ar…

Insilico Medicine, a global leader in artificial intelligence for drug discovery, announced the publication of a paper titled, "Deep learning enables rapid identification...

AstraZeneca agrees to buy US FDA Priority Review V…

AstraZeneca announced that it has agreed to buy a US Food and Drug Administration (FDA) Priority Review Voucher (PRV) for a total cash consideration of $95m from a subsid...

Boehringer Ingelheim eExpands KRAS cancer program …

Boehringer Ingelheim and Lupin Limited (Lupin) announced a licensing, development and commercialization agreement for Lupin's MEK inhibitor compound (LNP3794) as a potent...

Pfizer invests half billion dollars to advance sta…

Pfizer announced an additional half billion dollar investment for the construction of its state-of-the-art gene therapy manufacturing facility in Sanford, North Carolina...

FDA grants Fast Track designation for Farxiga in c…

AstraZeneca today announced that the US Food and Drug Administration (FDA) has granted Fast Track designation for the development of Farxiga (dapagliflozin) to delay the ...

The Pfizer Foundation invests in 20 organizations …

The Pfizer Foundation announced 20 grants* to help non-governmental organizations (NGOs), non-profits and social enterprises address critical health challenges related to...

Tagrisso approved in China as a 1st-line treatment…

AstraZeneca today announced that it has received marketing authorisation from China's National Medical Products Administration (NMPA) for Tagrisso (osimertinib) as a 1st-...