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FDA Updates for the Week of Aug. 14, 2023


It was a busy week for the FDA. Regulators approved several new therapies, including the first for a rare bone disorder, a combination product for metastatic prostate cancer and a long-acting botulinum toxin for cervical dystonia, as well as an accelerated approved for multiple myeloma. The agency also set review dates for three products, including for full approval of Tarpeyo for a rare kidney disease, an additional indication for Tibsovo for myelodysplastic syndromes and a combination antibiotic for complicated UTIs.

FDA Approves First Drug for Rare Bone Disorder.

The FDA has approved Sohonos (palovarotene) to reduce new abnormal bone formation in people living with fibrodysplasia ossificans progressiva (FOP), an ultra-rare genetic disease that transforms the body’s soft issue into bone. Sohonos is indicated to treat girls 8 years of age and older and boys 10 years of age or older.

FOP impacts less than an estimated 400 people in the United States and 900 people globally. By the age of 30 years old, the majority of people with FOP require a wheelchair and full-time caregiver assistance. Life expectancy is about 56 years, and the management of FOP has previously been limited to palliative care.

Developed by Ipsen, Sohonos targets the gamma subtype of retinoic-acid receptors, which are important regulators of skeletal development. It is designed to mediate the interactions between the receptors, growth factors and proteins within the retinoid signaling pathway to reduce new abnormal bone formation.

Sohonos is now available. The annual list price is $624,000 based for 5 mg dose per day, which a spokesperson for Ipsen said is based on the value that medicine provides, including clinical benefit, economic value, social and quality of life measures, prevalence of the condition and if treatment options are available. She said the price may vary because the treatment is variable for pediatric patients based on weight and dose-increases for flare-up episodes.

FDA Grants Accelerated Approval to Elrexfio for Multiple Myeloma.

The FDA has granted accelerated approval to Pfizer’s Elrexfio (elranatamab-bcmm) to treat adult patients with relapsed or refractory multiple myeloma (RRMM). It is indicated for patients who have had at least four prior lines of therapy.

Multiple myeloma is an aggressive and currently incurable blood cancer that affects plasma cells made in the bone marrow, and more than 35,000 new cases of multiple myeloma are diagnosed annually in the United States.

Elrexfio is a subcutaneously delivered B-cell maturation antigen (BCMA)-CD3-directed bispecific antibody immunotherapy. It binds to BCMA on myeloma cells and CD3 on T-cells, bringing them together and activating the T-cells to kill myeloma cells. It is the first BCMA-directed therapy in the United States with the option for every-other-week dosing after 24 weeks of weekly treatment.

Elrexfio will be available within the next few weeks. It will have a list price of $7,556 for the 44 mg vial and $13,051 for the 76 mg vial, or a monthly price of $41,500, which a spokesperson said is similar to other approved multiple myeloma therapies. The company expects the monthly price to be lower (about $26,000) as patients move to bi-weekly dosing.

FDA Approves Long-Acting Botulinum Toxin for Cervical Dystonia.

The FDA has approved the first therapeutic indication for Daxxify (daxibotulinumtoxinA-lanm) to treat adults with cervical dystonia in adults. Cervical dystonia is a chronic, debilitating disease in which the neck muscles contract involuntarily, causing abnormal movements, pain and awkward posture of the head and neck. It affects about 60,000 Americans.

Botulinum toxin injections are the standard of care for cervical dystonia, which works by relaxing the affected muscles and reducing spasming and stiffness. But the treatment effect gradually wears off, which is why patients need to be retreated on a regular basis.

Daxxify injection is a long-acting acetylcholine release inhibitor and neuromuscular blocking agent. Developed by Revance Therapeutics, Daxxxify was developed with a stabilizer that allows for longer treatment effect, between 15 weeks and 28 weeks. It will be available beginning in 2024 after a company’s sponsored education program, CD PrevU. A list price will be available when it is launched, according to a company spokesperson.

FDA Approves Combination Tablet for Metastatic Prostate Cancer.

The FDA has approved Janssen’s Akeega (niraparib and abiraterone acetate) tablet to treat patients with BRCA-positive metastatic castration-resistant prostate cancer (mCRPC). This is the first dual-action tablet formulation for these patients.

Metastatic castration-resistant prostate cancer is a cancer that has progressed despite medical or surgical treatment to lower testosterone. The most common metastatic sites are bones, followed by lungs and liver. About 10% to 50% of patients progress to mCRPC within three years of diagnosis, of which an estimated 10% to 15% harbor a BRCA mutation. Patients with mCRPC and HRR gene alterations are more likely to have aggressive disease, poor outcomes and a shorter survival time.

The combination of niraparib, a PARP inhibitor, and abiraterone acetate, a prodrug of abiraterone, a CYP17 inhibitor, targets two oncogenic drivers in patients with mCRPC: the androgen receptor axis and HRR gene alterations. It will be available in mid-September and have a list price of $18,750 for a 30-day supply, which a Janssen spokesperson said is comparable with other mCRPC PARP plus abiraterone therapies.

FDA Sets PDUFA Date for Full Approval of Tarpeyo.

The FDA has accepted the submission for the supplemental new drug application (sNDA) for Tarpeyo (budesonide) and granted the application priority review. The NDA is seeking full approval of Tarpeyo to reduce protein level in the urine of adults with primary IgA nephropathy (IgAN) at risk of rapid disease progression. The Prescription Drug User Fee Act (PDUFA) goal date is Dec. 20, 2023.

Primary immunoglobulin A nephropathy (IgAN) is a rare, progressive, chronic autoimmune disease that attacks the kidneys. Also called Berger’s disease, it can lead to progressive kidney damage. IgAN most often develops between late teens and late 30s. About 60,000 in the United States have IgAN, according to the National Kidney Foundation.

Develped by Calliditas Therapeutics, Tarpeyo was granted accelerated approval in December 2021. It is an oral, delayed release formulation of budesonide, a corticosteroid. Tarpeyo 4 mg has a list price of about $15,934 for a supply of 120 capsules, according to Drugs.com. The recommended dose is 16 mg once daily.

FDA Sets Review Date for Combination Antibiotic for Complicated UTIs.

FDA has accepted a new drug application (NDA) for cefepime-taniborbactam injection to treat adult patients with complicated urinary tract infections (cUTI), including patients who have pyelonephritis where the infection spreads to the kidneys. The FDA has granted Priority Review of the NDA with a Prescription Drug User Fee Act (PDUFA) target action date of Feb. 22, 2024.

Urinary tract infections are common, especially in women. But urinary infections in immunocompromised patients, mens, and those associated with fevers, stones, sepsis, urinary obstruction, catheters, or involving the kidneys are considered complicated infections. In the United States, there are more 626,000 hospital admissions a year for complicated urinary tract infections, a recent study found.

Cefepime, a fourth-generation cephalosporin, is a widely used beta-lactam antibiotic effective against susceptible gram-negative and gram-positive bacteria. Taniborbactam is a beta-lactamase inhibitor. Together the two have the potential to treat patients with serious bacterial infections caused by antibiotic resistant gram-negative bacteria. Patients with carbapenem-resistant strains of Enterobacterales infections had longer hospital lengths of stay, were less likely to be discharged home, and had a higher readmission rate, finds a separate study.

The combination therapy was developed by Everest Medicines, which has partnered with Venatorx Pharmaceuticals.

FDA Grants Priority Review for Tibsovo sNDA for Myelodysplastic Syndromes.

The FDA has granted priority review for the Servier’s supplemental new drug application (sNDA) for Tibsovo (ivosidenib) to treat patients with relapsed or refractory (R/R) myelodysplastic syndromes (MDS). It would be indicated to treat patients with isocitrate dehydrogenase 1 (IDH1) mutations. If approved, it would be the first approved targeted therapy for MDS patients with an IDH1 mutation. With priority review, the FDA provides a decision on an application within six months.

Myelodysplastic syndromes are disorders in which stem cells do not mature into healthy blood cells. In the United States, about 16,000 new cases of MDS are reported each year, and one-third of these patients will eventually progress to acute myeloid leukemia (AML). About 3.6% of MDS patients have an IDH1 mutation, which has been associated with worse overall outcomes.

Tibsovo is currently approved in the United States as a monotherapy to treat adults with IDH1-mutant relapsed or refractory AML and in monotherapy or in combination with azacitidine for adults with newly diagnosed IDH1-mutant AML who are 75 years old or older. It has a list price of $31,687 for a supply of 60 tablets, according to Drugs.com.

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