 to the FDA seeking approval for Skyrizi (risankizumab-rzaa), an interleukin-23 (IL-23) inhibitor, for the treatment of patients 16 years and older with moderate-to-severe Crohn’s disease. Approval is being sought for both the 600 mg intravenous induction and 360 mg subcutaneous maintenance therapy.

The submission is supported by safety and efficacy data from three phase 3 studies – ADVANCE, MOTIVATE and FORTIFY. In these trials, Skyrizi demonstrated improvements in clinical remission and endoscopic response as both induction and maintenance therapy. The overall safety findings in these studies were consistent with previous studies.

In the FORTIFY study, a significantly greater proportion of participants achieved the co-primary end points of endoscopic response and clinical remission with Skyrizi subcutaneous maintenance therapy at one year (52 weeks) for both assessed doses (360 mg or 180 mg), compared with those who were randomized to the withdrawal arm and received placebo. 

In the ADVANCE and MOTIVATE induction studies, a significantly greater proportion of patients with Crohn's disease treated with either dose of Skyrizi IV induction therapy (600 mg or 1200 mg) met the co-primary end points of clinical remission and endoscopic response at week 12 compared with placebo.

“While there have been advancements in care, many people with Crohn's disease do not achieve lasting remission,” Tom Hudson, senior vice president of research and development, chief scientific officer, AbbVie, said in a statement.

In April 2019, the FDA approved Skyrizi for the treatment of moderate-to-severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy. Phase 3 trials of Skyrizi in psoriatic arthritis, Crohn's disease, and ulcerative colitis are ongoing.

 is a chronic disorder that primarily affects the digestive system. This condition involves an abnormal immune response that causes excess inflammation. It most often affects the intestinal walls, particularly in the lower part of the small intestine and portions of the large intestine. Crohn’s disease is most common in western Europe and North America, where it has a prevalence of 100 to 300 per 100,000 people. More than half a million Americans are currently affected by this disorder.

Articles

In trials, Skyrizi demonstrated improvements in clinical remission and endoscopic response as both induction and maintenance therapy

AbbVie Submits Skyrisi to FDA for Crohn’s Disease

 priority review of the Biologics License Application (BLA) for the combination of relatlimab and Opdivo (nivolumab), administered as a single infusion, for the treatment of adult and adolescents with unresectable or metastatic melanoma. The FDA assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 19, 2022.

The BLA submission was based on the efficacy and safety results of the phase 2/3 RELATIVITY-047 trial, which demonstrated that the combination therapy achieved progression-free survival benefit of 10.1 months compared with 4.6 months for Opdivo alone.

“We believe that the relatlimab and nivolumab fixed-dose combination has the potential to improve outcomes for patients with metastatic or unresectable melanoma,” Jonathan Cheng, senior vice president and head of oncology development, Bristol Myers Squibb, said in a statement.

Relatlimab is the first LAG-3-blocking antibody to demonstrate a clinical benefit for patients with phase 3 data. Primary results from the RELATIVITY-047 trial were 

 during the American Society of Clinical Oncology (ASCO) Annual Meeting in June 2021. Adverse events leading to discontinuation of treatment in 14.6% of patient receiving the combination therapy versus 6.7% for those receiving Opdivo alone.

Lymphocyte-activation gene 3 (LAG-3) functions to control T-cell response, activation, and growth.

Melanoma is the fifth most common cancer, 

 to the National Institutes of Health. The NIH estimates that in 2021 more than 7,000 people will die from melanoma.

The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 19, 2022.

FDA Accepts BMS Application for Opdivo/Relatimab Combination for Advanced Melanoma

Denise Myshko is your editorial contact for the Formulary Watch. She joined Formulary Watch as editor is June 2021 from Oncology Live. Prior to that, she was managing editor of PharmaVOICE, a trade publication in the pharmaceutical industry. Email: dmyshko@mjhlifesciences.com

 Biogen and Samsung Bioepis’ Byooviz (ranibizumab-nuna) as the first biosimilar to Genentech’s Lucentis (ranibizumab injection) for the treatment of several eye diseases and conditions, including neovascular (wet) age-related macular degeneration, a leading cause of vision loss and blindness for Americans over the age of 65. Byooviz is also approved to treat macular edema following retinal vein occlusion (blockage of veins in the retina) and myopic choroidal neovascularization, a vision-threatening complication of myopia (nearsightedness).

Byooviz is an anti-vascular endothelial growth factor (VEGF) therapy that prevents vision loss in patients with retinal vascular disorders, which can cause irreversible blindness or visual impairments in adults in the United States.

The FDA approval of Byooviz was based on a totality of evidence including analytical, nonclinical data, and clinical data. In a phase 3 study, the efficacy, safety, pharmacokinetics, and immunogenicity of Byooviz was compared with Lucentis in patients with wet AMD.

“Today’s approval provides another treatment option for millions of people whose vision is impaired and is another step forward in our commitment to provide access to safe, effective and high-quality biological products,” Sarah Yim, M.D., director of the Office of Therapeutic Biologics and Biosimilars in the FDA’s Center for Drug Evaluation and Research, said in a statement.

There are two types of AMD—dry and wet. While dry AMD is more common, wet AMD leads to vision loss at a faster rate. Both macular edema and myopic choroidal neovascularization can also lead to visual impairment. About 11 million people 

 with AMD in the United States alone, with a global prevalence of 170 million.

Byooviz was approved in the European Union (EU) on August 18, 2021, and the United Kingdom on August 31, 2021.

BYOOVIZ is Samsung Bioepis’ fifth biosimilar approved in the United States, following the approval of Renflexis (infliximab-abda), a biosimilar of Remicade approved in April 2017 to treat several autoimmune diseases including Crohn’s and rheumatoid, Ontruzant (trastuzumab-dttb), a biosimilar of Herceptin approved in January 2019 to treat HER2-positive breast and gastric cancer, Eticovo (etanercept-ykro), a biosimilar of Enbrel approved in April 2019 to treat arthritis and plaque psoriasis, and Hadlima (adalimumab-bwwd), a biosimilar of Humira approved in July 2019 to treat arthritis and psoriasis.

Biogen/Samsung Bioepis’ Byooviz is a biosimilar of Genentech’s Lucentis. 

FDA Approves First Biosimilar to Treat Macular Degeneration 

The Centers for Medicare & Medicaid Services (CMS) 

 a permanent J-code (J2406) for Melinta Therapeutics’ Kimyrsa (oritavancin), a lipoglycopeptide antibiotic, effective October 1, 2021. This code allows the product to be reimbursed when dispensed in the outpatient setting. Kimyrsa is approved to treat acute bacterial skin and skin structure infections in a single, one-hour, 1,200 mg infusion. The FDA approved the therapy on March 12, 2021, for the treatment of adult patients with Gram-positive infections, including methicillin-resistant Staphylococcus aureus (MRSA).

At the same time, CMS has granted transitional pass-through status for Kimyrsa in the hospital outpatient department setting. Pass-through status is intended to protect Medicare beneficiaries’ access to innovative drugs and ensures that Kimyrsa will be reimbursed at average sales price for three years. Drugs with pass-through status are not subject to the payment reduction applicable to drugs purchased through the 340B program.

“We intentionally designed Kimyrsa to provide patients with more flexibility and accessibility in acute bacterial skin and skin structure infection treatment. These achievements are one more significant step toward our vision that all patients who need our therapies will be able to receive them,” Christine Ann Miller, Melinta’s president and chief executive officer, said in a statement.

Bacterial skin and skin structure infections affect about 14 million patients in the United States each year.

Effective October 1, 2021, CMS will reimburse for the infusion product when dispensed in the outpatient setting.

CMS Assigns J-Code for the Antibiotic Kimyrsa 

 Exelixis’ Cabometyx (cabozantinib) for the treatment of adult and pediatric patients 12 years of age and older with locally advanced or metastatic differentiated thyroid cancer that has progressed following treatment with a vascular endothelial growth factor receptor (VEGFR)-targeted therapy and who are resistant to radioactive iodine treatment.

This is more than two months ahead of the Prescription Drug User Fee Act (PDUFA) target action date of December 4, 2021. DTC is the most common type of thyroid cancer in the United States, and patients who are resistant to radioactive iodine treatment face a poor prognosis.

The approval is based on results from COSMIC-311, a phase 3 pivotal trial. At an interim analysis, Cabometyx reduce the risk of progression or death. At a follow-up analysis, the median progression-free survival was 11.0 months.

Serious adverse events seen in at least 5% of patients included hypertension, fatigue, diarrhea, and palmar-plantar erythrodysesthesia, which is a side effect that can cause redness, swelling, and pain in the hands and feet.

“Before today, patients with radioactive iodine-refractory differentiated thyroid cancer who have progressed following prior VEGFR-targeted therapy were facing aggressive disease and no standard treatment option,” Marcia S. Brose, M.D., Ph.D., chief, cancer center operation, Sidney Kimmel Cancer Center at Jefferson Torresdale Hospital, co-director, community based clinical trials, Sidney Kimmel Cancer Center at Thomas Jefferson University, and principal investigator of COSMIC-311, said in a statement.

In February 2021, the FDA granted Breakthrough Therapy Designation to Cabometyx for this indication.

Cabometyx is also approved for the treatment of patients with renal cell carcinoma (RCC), hepatocellular carcinoma who have been previously treated with Nexavar (sorafenib); and for patients with advanced RCC as a first-line treatment in combination with Opdivo (nivolumab).

The American Cancer Society estimates that 44,280 new cases of thyroid cancer will be diagnosed in the United States this year. Most (73%) of those cases will be in women. About 2,200 people will die from the disease.

The approval comes more than two months ahead of its PDUFA target date of December 4, 2021.


FDA Approves Cabometryx for Thyroid Cancer

Formulary Watch Drug Shortage Tracker — September 19, 2021

The FDA’s Vaccines and Related Biological Products Advisory Committee voted 16-2 against full approval for the use of Pfizer/BioNTech’s COVID-19 vaccine, Comirnaty, in people over the age of 16. But they did vote unanimously to provide an emergency use authorization of the vaccine as a booster after six months for those over the age of 65 and those at high risk of severe COVID-19.

Committee members recognized research that showed waning immunity from the COVID vaccines over time, but they indicated there was not enough data to support a third dose in the general population. They were concerned about the safety of the third dose of the vaccine, specifically heart inflammation, and they wanted to see data specifically on this area of safety.

Materials the FDA released before the meeting included 

 of real-world data in Israel that found a third dose of the vaccine boosted vaccine effectiveness to the level initially seen after the second dose. Investigators found that the rate of confirmed infection was lower in the booster group than in the nonbooster group by a factor of 11.3, and the rate of severe illness was 7.5 cases per 100,000 person-days.

The third dose of the Pfizer vaccine in Israel was approved on July 30, and investigators analyzed data through Aug. 31, 2021. This study was 

FDA advisory committee members were concerned about myocarditis in younger people given the third dose. Investigators in Israeli study found that out of about 2 million booster doses, a small number of people experienced serious adverse events.

In the United States, safety surveillance reports received by FDA and CDC identified serious risks for myocarditis and pericarditis following administration of the first two doses of the vaccine, with higher rates seen in men under the age of 40 with the highest rates among male adolescents 16 and 17 years of age, according to a 

. An FDA analysis of the Optum database estimated an excess risk approaching 200 cases per million vaccinated males 16 and 17 years of age, but information was not available on long-term outcomes related to myocarditis.

When polled, all committee members agreed when polled to extending the EUA booster to those at risk for occupational exposure, including health care workers.

 the EUA for a booster of the Pfizer vaccine to those at least 12 years of age who have undergone an organ transplant, or who have conditions that lead to a similar level of level of compromised immune system. This authorization is separate and distinct from the booster dose reviewed by the committee.


The vaccines advisory committee voted late on Friday, September 17, 2021, to provide emergency authorization of Pfizer’s COVID-19 vaccine as a booster for those over the age of 65, as well as for those at high risk of developing severe disease. 


FDA Advisory Committee Supports COVID-19 Boosters for Some People

 applications to the FDA and the EMA for Rinvoq (upadacitinib) for the treatment of adults with moderate-to-severe ulcerative colitis. The applications are supported by data from two phase 3 induction studies and one maintenance study.

In these studies, more patients treated with Rinvoq achieved the primary endpoint of clinical remission and all secondary end points compared with placebo with 45 mg once daily at week eight for the induction studies, and with 15 mg and 30 mg once daily at 52 weeks for the maintenance study.

 of Rinvoq 15 mg or 30 mg were consistent with the safety profile observed in the phase 3 induction studies in ulcerative colitis, as well as in previous studies across indications. No new safety risks were identified.

“Many people continue to struggle with symptoms associated with their ulcerative colitis, such as fatigue, bowel urgency, bloody diarrhea and abdominal pain,” Tom Hudson, M.D., senior vice president of research and development, chief scientific officer, AbbVie, said in a statement. "Upadacitinib has the potential to be an important new treatment option for people with ulcerative colitis, who want to address these challenging and disruptive symptoms. We look forward to working with regulatory authorities and hope to bring upadacitinib to people with ulcerative colitis as quickly as possible."

Ulcerative colitis is a chronic inflammatory bowel disease of the large intestine. In 2015, an estimated 1.3% of U.S. adults (3 million) reported being diagnosed with inflammatory bowel disease, either Crohn’s disease or ulcerative colitis, 

Rinvoq is a JAK inhibitor that is being studied in several immune-mediated inflammatory diseases. Rinvoq 15 mg is approved in the United States for adults with moderate to severe rheumatoid arthritis. It is approved in the EU for adults with moderate to severe active rheumatoid arthritis, adults with active psoriatic arthritis, adults with active ankylosing spondylitis (AS), and adults and adolescents 12 years and older with moderate to severe atopic dermatitis (AD). Rinvoq 30 mg is approved in the EU for adults with moderate to severe atopic dermatitis under age 65.

Related: FDA’s New Warning Includes Xeljanz and other JAK Inhibitors

Rinvoq achieved primary and secondary end points compared with placebo.


AbbVie Submits Rinvoq to FDA and EMA for Ulcerative Colitis

 all remaining lots of Chantix (varenicline) 0.5 mg and 1 mg tablets because of the presence of a nitrosamine, which may be associated with an increased risk of cancer.

Chantix is a treatment to help patients quit smoking and is intended for short term use. The products were distributed nationwide to wholesalers and distributors in the United States, U.S. Virgin Islands, and Puerto Rico from May 2019 to September 2021.

The NDC, lot numbers, and expiration date affected can be found 

 made over the last few months when 16 lots of Chantix had been recalled.

Pfizer officials say they are making this move as precaution since other options are now available. Last month, the FDA 

 the first generic version of Chantix. Developed by Par Pharmaceutical, which is part of Endo International, the generic varenicline tablet was approved August 11, 2021. The generic will come in doses of 0.5 mg and 1 mg.

Additionally, In July after Pfizer’s initial recalls, Apotex Corp., in coordination with the FDA, 

 temporary importation of Apo-Varenicline (varenicline tartrate) 0.5 mg and 1 mg tablets into the U.S. market. Apo-Varenicline is the varenicline tartrate product approved by Health Canada as a generic equivalent to Champix tablets manufactured by Pfizer Canada. Champix is the brand name of Chantix in Canada.


To date, Pfizer has not received reports of adverse events related to this recall, but is making the move as a precaution. 



Pfizer Recalls All Lots of Smoking Cessation Drug Chantix

 Eli Lilly’s emergency use authorization (EUA) for bamlanivimab 700 mg and etesevimab 1400 mg administered together to include post-exposure prophylaxis.

The neutralizing antibodies can now be used together to treat high-risk individuals 12 years of age and older who have not been fully vaccinated against COVID-19 or are not expected to mount an adequate immune response to complete vaccination, and have been exposed to someone infected with SARS-CoV-2.

 for the U.S. government to purchase 388,000 doses of etesevimab to complement doses of bamlanivimab previously purchased by the U.S. government, with about 200,000 doses expected to ship third quarter of 2021 and the remaining to be shipped in fourth quarter. This transaction is expected to generate about $330 million in revenue in the second half of 2021, according to a Lilly statement.

"The recent increase in COVID-19 cases has caused a substantial rise in the utilization of monoclonal antibody drugs, particularly in areas of the country with low vaccination rates," Daniel Skovronsky, M.D., Ph.D., Lilly's chief scientific and medical officer, and president of Lilly Research Laboratories, said in a statement. "Lilly developed bamlanivimab and etesevimab for administration together, in anticipation of variants such as the highly contagious Delta variant, which currently accounts for more than 98 percent of all identified COVID-19 cases in the United States."

The expanded authorization is based on data from BLAZE-2, a study conducted in partnership with the National Institute of Allergy and Infectious Diseases and the COVID-19 Prevention Network that enrolled residents and staff at long-term care facilities across the United States. In this placebo-controlled phase 3 study, bamlanivimab reduced the risk of contracting symptomatic COVID-19 by up to 80% in nursing home residents and up to 57% among residents and staff of long-term care facilities.

Bamlanivimab and etesevimab together are authorized for the treatment of mild-to-moderate COVID-19 in adults and adolescents (12 years of age) with positive results of direct SARS-CoV-2 viral testing. But the combination is not authorized for use in patient who are hospitalized or who need oxygen therapy.

Bamlanivimab is a recombinant, neutralizing human IgG1 monoclonal antibody directed against the spike protein of SARS-CoV-2. It was designed to block viral attachment and entry into human cells. Etesevimab is a recombinant fully human monoclonal neutralizing antibody that specifically binds to the SARS-CoV-2 surface spike protein receptor and can block the binding of the virus to the ACE2 host cell surface receptor.

The combination of bamlanivimab/etesevimab can now be given to both treat and prevent COVID-19 infection after exposure.

FDA Expands EUA for Lilly’s COVID-19 Monoclonal Antibody

 the emergency use authorization (EUA) for the Pfizer-BioNTech COVID-19 vaccine to allow for use of a single booster dose for:

People through 64 years of age at high risk of severe COVID-19; and

People 18 through 64 years of age at risk for occupational exposure to COVID-19.

The FDA authorized the booster to be given at least six months after completion of the primary vaccination.

“This pandemic is dynamic and evolving, with new data about vaccine safety and effectiveness becoming available every day. As we learn more about the safety and effectiveness of COVID-19 vaccines, including the use of a booster dose, we will continue to evaluate the rapidly changing science and keep the public informed,” Acting FDA Commissioner Janet Woodcock, M.D., said in a statement.

This authorization comes less than a week after the FDA’s Vaccines and Related Biological Products Advisory Committee voted 16-2 against full approval for the use of Pfizer’s vaccine, now called Comirnaty, in people over the age of 16. But they did vote unanimously to provide an emergency use authorization of the vaccine as a booster after six months for those over the age of 65 and those at high risk of severe COVID-19.

Read more about the committee’s decision

Pfizer and BioNTech continue to supply the vaccine under their existing 

 with the U.S. government, which continues through April 2022. Executives from the companies do not expect the introduction of booster doses in the United States to impact the existing supply agreements in place with governments and international health organizations.

We will continue to monitor new SARS-CoV-2 strains, to be prepared for potential emerging escape variants,” Ugur Sahin, M.D., CEO and co-founder of BioNTech, said in a statement. “We and our collaboration partner have submitted booster data to other regulatory agencies around the world. We are simultaneously working to expand access to our vaccines globally.”

The U.S. Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices (ACIP) will meet to discuss a potential recommendation for the use and rollout of boosters.


People over the age 65 and those at high risk of severe COVID-19, including health care workers and those with occupational exposure, are eligible for the booster. 


FDA Issues EUA for Booster of Pfizer’s COVID-19 Vaccine

Par Pharmaceutical, part of Endo International, has 

 the generic version of Pfizer's Chantix (varenicline), 0.5 mg and 1 mg tablets. Developed by Par Pharmaceutical, which is part of Endo International, the generic varenicline tablet was approved by the FDA August 11, 2021.

 all remaining lots of Chantix due to the presence of a nitrosamine, N-nitroso-varenicline, which may be associated with a potential increased cancer risk in humans.

Dr. Reddy’s launches generic of Librax.

 chlordiazepoxide hydrochloride and clidinium bromide

capsules, a generic version of Librax (chlordiazepoxide hydrochloride and clidinium bromide). It is approved to treat gastrointestinal disorders, such as peptic ulcer, irritable bowel syndrome (irritable colon, spastic colon, mucous colitis), and acute enterocolitis. It is available in one strength of 5 mg/2.5 mg capsules in bottle count sizes of 100.

FDA approves generic clindamycin phosphate foam.

 FDA approval for clindamycin phosphate foam, 1%, the generic version of Mylan’s Evoclin Foam, 1%, an antibiotic that is used to treat acne.

According to IQVIA sales data for the 12-month period ending July 2021, the Evoclin Foam, 1%, market achieved annual sales of about $12.0 million

 the abbreviated new drug application of Milla Pharmaceuticals’ generic version of sodium acetate injection 2 MEQ/mL as a source of sodium as an addition to large volume intravenous fluids to prevent or correct hyponatremia in patients with restricted or no oral intake. It is also used as an additive for preparing specific intravenous fluid formulas when the needs of the patient cannot be met by standard electrolyte or nutrient solutions.

The company, a subsidiary of the Alter Pharma Group, has announced that it has launched this product.

Milla Pharmaceuticals has established its first commercial sale through its exclusive partner, Woodward Pharma Services, and has secured 180-days of market exclusivity. The FDA has recently 

 sodium acetate Injection 2MEQ/mL on their its drug shortages list.

Dr. Reddy’s has also launched a generic of Librax. Recent generic approvals include clindamycin phosphate foam and sodium acetate.

Par Launches Generic of Smoking Cessation Drug Chantix

 Incyte’s Opzelura (ruxolitinib) cream for the short-term and non-continuous chronic treatment of mild-to-moderate atopic dermatitis in patients 12 years of age and older whose disease is not adequately controlled with topical prescription therapies. Opzelura is the first and only topical formulation of a JAK inhibitor approved in the United States. Research shows dysregulation of the JAK-STAT pathway contributes to key features of atopic dermatitis such as itch, inflammation, and skin barrier dysfunction.

The FDA approval was based on data from two phase 3 studies (TRuE-AD1 and TRuE-AD 2) evaluating the safety and efficacy of Opzelura in more than 1,200 adolescents and adults. Results from the studies showed patients experienced significantly clearer skin and itch reduction when treated with Opzelura cream 1.5% twice daily, compared with a non-medicated cream. Significantly more patients treated with Opzelura experienced a clinically meaningful reduction in itch from baseline at Week 8.

The most common adverse reactions were nasopharyngitis, diarrhea, bronchitis, ear infection, eosinophil count increased, urticaria, and tonsillitis.

“Atopic dermatitis is a chronic immune-mediated disease that can be challenging to manage. Many patients do not respond well to existing treatments and have uncontrolled disease,” Jonathan Silverberg, M.D., Ph.D., M.P.H., associate professor of dermatology and director of clinical research and contact dermatitis at The George Washington University School of Medicine and Health Sciences, said in a statement.

Opzelura is a novel cream formulation of Incyte’s selective JAK1/JAK2 inhibitor ruxolitinib.

Atopic dermatitis is a chronic skin disease affecting more than 21 million people aged 12 years and older in the United States and is characterized by inflammation and itch.

Opzelura is the first and only topical formulation of a JAK inhibitor approved in the United States. 

FDA Approves Topical JAK inhibitor for Atopic Dermatitis

 to Genmab/Seagen’s Tivdak (tisotumab vedotin-tftv), the first approved antibody-drug conjugate (ADC) for the treatment of adult patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy. Tivdak is approved under the FDA’s Accelerated Approval Program based on tumor response and the durability of the response. Approval is contingent upon confirmatory trials.

The approval is based on the phase 2 innovaTV 204 trial, which evaluated 101 patients. Results from the trial showed a 24% objective response rate, and the median duration of response was 8.3 months.

The most common adverse reactions were hemoglobin decreased (52%), fatigue (50%), lymphocytes decreased (42%), nausea (41%), peripheral neuropathy (39%), alopecia (39%), epistaxis (39%), conjunctival adverse reactions (37%), hemorrhage (32%), and leukocytes decreased (30%).

These data were featured in a late-breaking proffered paper oral presentation at 

 in April 2021. A global, randomized phase 3 clinical trial (innovaTV 301) is under way and is intended to support global registrations.

“Once recurrent or metastatic cervical cancer progresses, there is a need for more options for these patients,” Robert L. Coleman, M.D., chief scientific officer, US Oncology Research and lead investigator of the innovaTV 204 clinical trial, said in a statement. “This is an important development for patients with recurrent or metastatic cervical cancer.”

Tivdak is an ADC composed of Genmab’s human monoclonal antibody directed to tissue factor and Seagen’s ADC technology that uses a protease-cleavable linker that covalently attaches the microtubule-disrupting agent monomethyl auristatin E to the antibody. Nonclinical data suggests that the anticancer activity of the therapy is due to the binding of the ADC to tissue factor-expressing cancer cells.

In 2018, there were an estimated 293,394 women living with cervical cancer in the United States. The National Institutes of Health 

 there will be an additional 14,480 cases this year and an estimated 4,290 deaths from cervical cancer.

Tivdak is the first approved antibody-drug conjugate for the treatment of adult patients with recurrent or metastatic cervical cancer.

FDA Approves Tivdak for Advanced Cervical Cancer

 for filing Marinus Pharmaceuticals’ new drug application (NDA) for the use of ganaxolone in the treatment of seizures associated with CDKL5 deficiency disorder, a rare, genetic epilepsy. The NDA was granted Priority Review designation and the FDA assigned a Prescription Drug User Fee Act (PDUFA) action date of March 20, 2022.

The application is supported by data from the Marigold study, a phase 3 trial in 101 patients. Patients treated with ganaxolone showed a 30.7% median reduction in 28-day major motor seizure frequency, compared with a 6.9% reduction for those receiving placebo. Patients in the open-label extension study treated with ganaxolone for at least 12 months experienced a median 49.6% reduction in major motor seizure frequency.

Ganaxolone received orphan drug designation in 

 and a Rare Pediatric Disease designation for CDKL5 deficiency disorder in 

“The FDA’s acceptance of our NDA submission is an important step toward potentially bringing the first approved therapy specifically for treatment of seizures associated with CDD—a devastating disorder with high unmet medical need—to families and healthcare providers,” Scott Braunstein, M.D., chief executive officer of Marinus Pharmaceuticals, said in a statement. “We believe that ganaxolone has the potential to provide meaningful clinical benefit for patients and we look forward to working closely with the FDA during the review process.”

 that is caused by a mutation of the cyclin‑dependent kinase‑like 5 (CDKL5) gene, located on the X chromosome. CDKL5 deficiency disorder is characterized by early‑onset, difficult‑to‑control seizures and severe neuro‑developmental impairment. Currently, there are no therapies approved specifically for CDD. The incidence 1 in 40,000 to 60,000 newborns. About 90% of those diagnosed with CDKL5 deficiency disorder are girls.

The FDA assigned a PDUFA action date of March 20, 2022, for ganaxolone.


FDA Issues EUA for Booster of Pfizer’s COVID-19 Vaccine

Par Launches Generic of Smoking Cessation Drug Chantix

FDA Approves Topical JAK inhibitor for Atopic Dermatitis

FDA Approves Tivdak for Advanced Cervical Cancer

FDA has Accepted Application for Rare Epilepsy