Most Important FDA Novel Drug Approvals of 2021, So Far

FDA Greenlights Biogen’s AZ Drug, Aduhelm

In June, FDA officially approved Aduhelm, Biogen’s Alzheimer’s disease drug. This approval marked the first Alzheimer’s drug approval in nearly 20 years and the first to address cognitive decline in those with the illness.

The approval came after long speculation about the drug’s efficacy. Biogen pulled the drug in 2019 after Phase 3 trial results showed that the drug administered as a monthly infusion was not better at slowing memory loss and cognitive impairment than placebo. 

FDA Approves Duchenne Muscular Dystrophy Drug 

In February, FDA approved Amondys 45 injection to treat Duchenne muscular dystrophy in patients who have confirmed mutation of the gene that is amenable to exon 45 skipping. 

The agency based its approval on an increase in dystrophin production in skeletal muscle seen in patients treated with therapy. 

This is the first FDA-approved targeted treatment for patients with this type of mutation.

FDA Approves ADHD Medication for Children 

In March, FDA approved a first-of-its-kind ADHD medication, Azstarys, for children ages six and older. 

The once-daily capsule contains serdexmethylphenidate (SDX) and immediate-release dexmethylphenidate (d-MPH). This is the first ADHD medication to combine these two ingredients. 

A Phase 3 trial showed that Azstarys improved attention and behavior in children six to 12 years of age better than placebo. 

FDA OKs First Non-Azole Treatment for Vaginal Yeast Infection

In June, FDA approved Brexafemme to treat vaginal yeast infections. This is FDA’s first approval of a new antifungal medication in over 20 years. 

Brexafemme comes as a 150 milligram oral tablet. An infected individual can take two tablets (300 milligrams) by mouth, and then 12 hours later, take another two tablets.

FDA Approves Bylvay for Rare Genetic Disorder

FDA recently approved Albireo Pharmaceuticals Bylvay for pruritus in progressive familial intrahepatic cholestasis (PFIC), a rare genetic disorder that causes progressive liver disease. 

Bylvay is a non-systemic ileal bile acid transport inhibitor (IBATi) that does not require refrigeration and is easily administered as a once-daily capsule or sprinkled onto soft foods. It is the first drug approved for pruritus in all subtypes of PFIC. 

ViiV Healthcare’s HIV Treatment for Adults Gets FDA Approval

In January, FDA approved ViiV Healthcare’s Cabenuva, the first and only complete long-acting injectable HIV treatment for positive adults.

Providers can administer the drug to HIV-positive adults once a month and will replace the current treatment regimen in those virologically suppressed with no history of treatment failure and with no known or suspected resistance to either cabotegravir or rilpivirine. 

FDA Approves Drug to Prevent Damage from Chemotherapy

FDA approved G1 Therapeutics’ Cosela, a therapy intended to reduce the frequency of chemotherapy-induced bone marrow suppression in adults for extensive-stage small-cell lung cancer. 

Cosela is the first FDA-approved therapy to provide protection from chemotherapy-induced myelosuppression.

FDA OKs Paroxysmal Nocturnal Hemoglobinuria Drug  

In May, FDA approved Empaveli injection to treat adults with paroxysmal nocturnal hemoglobinuria (PNH).

PNH is a rare and life-threatening blood disease. Empaveli is the first PNH treatment that binds to compliment protein C3, FDA stated in the announcement. 

In a study, patients that received treatment with Empaveli experienced increase in hemoglobin of 2.4 g/dL. And patients in this group also had an average decrease in their hemoglobin of 1.5 g/dL. 

FDA Approves Add-On Therapy for Severely High Cholesterol 

In February, FDA approved Evkeeza injection as an add-on treatment for patients 12 years of age and older with homozygous familial hypercholesterolemia. This genetic condition causes severely high blood pressure.

Sanofi’s Sleeping Sickness Drug Gets Nod from FDA

FDA recently approved Sanofi’s Fexinidazole, the first all-oral treatment for both stages of the Trypanosoma brucei gambiense form of sleeping sickness. 

The company developed the medication as part of a partnership between the non-profit research and development organization Drugs for Neglected Disease initiative (DNDi). 

FDA Approves Renal Cell Carcinoma Drug for Adults 

FDA recently approved Fotivda, a kinase inhibitor for adult patients with relapsed or refractory advanced cell carcinoma following two or more prior systemic therapies.

In a randomized, open-label trial, Fotivda elicited an overall survival rate of 18 percent, while chemotherapy drug sorafenib elicited overall survival of eight percent.

FDA OKs Immunotherapy Jemperli for Endometrial Cancer 

FDA recently granted accelerated approval to GSK’s Jemperli for patients with recurrent or advanced endometrial cancer that progressed on or following prior treatment with a platinum-containing chemotherapy.

Nearly 42.3 percent of patients who received Jemperli in a multi-cohort trial experienced complete or partial response. For about 93 percent of responders, the response lasted for six months or more.

FDA Approves Keytruda for Triple-Negative Breast Cancer Patients 

FDA recently approved Merck’s anti-PD-1 therapy, Keytruda, to treat patients with high-risk, early-stage triple-negative breast cancer (TNBC) in combination with chemotherapy.

The agency based its approval on the Phase 3 KEYNOTE-522 trial, which showed that Keytruda in combination with chemotherapy before surgery and continued as a single agent after surgery, substantially prolonged event-free survival (EFS) in TNBC patients compared to chemotherapy alone. 

FDA Approves Tablets for Complications of Chronic Kidney Disease

FDA recently approved Bayer’s Kerendia to reduce complications of chronic kidney disease associated with type 2 diabetes in adult and pediatric patients. 

FDA based its approval on a placebo-controlled study of 5,674 patients with chronic kidney disease associated with type 2 diabetes. The patients randomly received Kerendia (2,8333) or placebo (2,841)

First Oral Therapy for Lupus Nephritis Gets the Go Ahead

In January, FDA approved Lupkynis in combination with a background immunosuppressive therapy regimen to treat adult patients with active lupus nephritis. 

This is the first FDA-approved oral therapy for the disease. In previous trials, patients who received Lupkynis in combination with standard of care were over twice as likely to achieve renal response and decreased urine protein creatinine ratio. 

FDA OKs Lybalvi for Adults with Schizophrenia and Bipolar I Disorder

In June, FDA approved Alkermes Lybalvi to treat adults with schizophrenia and bipolar I disorder, as a the acute treatment of manic or mixed episodes, or an adjunct to lithium or valproate. 

In the ENLIGHTEN-2 study, the once-daily, oral atypical antipsychotic demonstrated efficacy, safety, and tolerability in patients with schizophrenia. Alkermes expects to make LYBALVI available for patients in the fourth quarter of 2021. 

FDA Approves Combination Oral Contraceptive 

FDA recently approved Nextstellis, a combination oral contraceptive birth control and the first type of estrogen approved in over 50 years. 

Nextstellis is a COC that contains 3 mg of drospirenone and 14.2 mg of estetrol. The pills should be taken simultaneously every day in the order instructed on the blister card.

FDA Approves Injection for Molybdenum Cofactor Deficiency Type A

FDA recently approved Nulibry to reduce the risk of death due to Molybdenum Cofactor Deficiency Type A.

Molybdenum Cofactor Deficiency Type A is a metabolic disorder that causes intractable seizures, brain injury, and death. This is the first FDA approval for a therapy to treat this disease. 

FDA Oks Infusion for Pompe Disease

FDA recently approved Nexviazyme for intravenous infusion to treat patients one year of age and older with late-onset Pompe disease.

Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on people’s lives. The approval brings patients with Pompe disease another enzyme replacement therapy option. 

FDA Grants Accelerated Approval for Multiple Myeloma Drug 

FDA recently granted accelerated approval to melphalan Pepaxto in combination with dexamethasone for adults with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy.

In the HORIZON trial, patients who received the combination treatment elicited an overall response rate of 23.7 percent and a median duration of response of 4.2 months. 

FDA Approves Modulator for Multiple Sclerosis 

FDA recently approved a once-daily oral selective sphingosine-1-phosphate receptor modulator, Ponvory, to treat adults with relapsing forms of multiple sclerosis (MS).

The agency based its approval on a two-year Phase 3 clinical trial in which Ponvory, 20 milligram demonstrated superior efficacy in reducing annual relapse by 30.5 percent compared to Aubagia 14 milligrams in patients with relapsing MS. 

FDA Approves PET Imaging Prostate Cancer Drug for Men 

FDA recently approved Pylarify, a drug for positron emission tomography (PET) imaging of prostate-specific membrane antigen (PSMA) positive lesions in men with prostate cancer.

Specifically, the agency indicated Pylarify for patients with suspected prostate cancer metastasis who are potentially curable by surgery or other therapy or for patients with suspected prostate cancer recurrence based on elevated serum prostate-specific antigen levels. 

FDA Approves Qelbree for ADHD

FDA recently approved Supernus Pharmaceuticals’ Qelbree to treat ADHD in individuals six to 17 years of age.

The medication, taken once a day, is the first novel non-stimulant approval in a decade. FDA based its approval on four Phase 3 trials that included more than 1,000 children six to 17 years of age. 

FDA OKs Drug for Chronic Graft-Versus-Host Disease 

FDA recently approved Rezurock to treat adult and pediatric patients 12 years of age and older with chronic graft-versus-host disease (cGVHD) after the failure of at least two prior lines of systemic therapy.

This treatment is the first and only approved small molecule inhibitor of ROCK2, a signaling pathway that modulates inflammatory responses and fibrotic processes.

FDA Approves Rybrevant for Non-Small Cell Lung Cancer Patients 

In May, FDA approved Janssen’s Rybrevant as the first targeted therapy for patients with subset of non-small cell lung cancer. 

The agency approved the therapy based on a study of 81 patients with non-small cell lung cancer and EGFR exon 20 insertion mutations whose disease had progressed during or after platinum-based chemotherapy.

FDA OK Key Component for Common Childhood Cancer Treatment

FDA recently approved Jazz Pharmaceutical’s Rylaze as a component of a chemotherapy regimen to treat the most common childhood cancer

Specifically, Rylaze was approved to treat lymphoblastic leukemia and lymphoblastic lymphoma in adult and pediatric patients allergic to E. coli-derived asparaginase products commonly used for cancer treatment.

FDA Approves Lupus Erythematosus Drug for Adults

FDA recently approved AstraZeneca’s Saphnelo, a type I interferon receptor antagonist to treat moderate to severe systemic lupus erythematosus in adult patients receiving standard therapy. 

The agency based its approval on Phase 2 and Phase 3 trials, which showed that patients treated with Saphnelo experienced a reduction in disease activity across organ systems, including skin and joints. 

The patients also further achieved sustained reduction in oral corticosteroid use compared to placebo.

FDA Grants Accelerated Approval for KRAS G12C Drug 

In June, FDA granted accelerated approval to Amgen’s Lumakras, the first treatment for adult patients with non-small cell lung cancer whose tumors have a specific type of genetic mutation and who have received at least one prior systemic therapy.

The agency specifically approved Lumakras for tumors with KRAS G12C, a type of mutation in a group of genes that help regulate cell growth and division. 

This is the first approved targeted therapy for tumors with any type of KRAS mutation.

Non-Small Cell Lung Cancer Drug Gets Accelerated Approval

FDA granted accelerated approval to Tepmetko for adult patients with metastatic non-small cell lung cancer harboring mesenchymal-epithelial transition (MET) exon 14 skipping alterations.

The agency based its decision on results from the VISION trial, which found that patients who received Tepmetko experienced an overall response rate of 42 percent, with a median response duration of 10.8 months. 

FDA Grants Accelerated Approval for Metastatic Cholangiocarcinoma Drug 

In May, FDA granted accelerated approval to Truseltiq, a kinase inhibitor for adults with previously untreated, unresectable locally advanced, or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 fusion. 

A multi-center open-label trial found that patients who received Truseltiq experienced an overall response rate of 23 percent, with one complete response and 24 partial responses.

Median duration of response was five months and among the 23 responders, eight patients maintained the response for six months or more.

FDA Gives Accelerated Approval to Kinase-Inhibitor Ukoniq

In February, FDA granted accelerated approval to Ukoniq, a kinase inhibitor including PI3K-delta and casein kinase CK1-epsilon, for two indications. 

The first indication is for patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least one prior anti-CD20-based regime. And the second indication is for adult patients with relapsed or refractory follicular lymphoma (FL) who have received at least three prior lines of systemic therapy.

FDA OKs Merck’s Drug to Reduce Cardiovascular Death in CHF Patients

In January, FDA Approved Merck’s Verquvo to reduce risk of cardiovascular death and heart failure hospitalization in adults with symptomatic chronic heart failure and ejection fraction less than 45 percent. 

The soluble guanylate cyclase stimulator is the first treatment for chronic heart failure explicitly approved for patients following hospitalization for heart failure or outpatient IV diuretics. 

FDA Approves Zegalogue for Severe Hypoglycemia in Diabetes Patients 

FDA approved Zegalogue to treat severe hypoglycemia in individuals with diabetes six years of age and older.

The agency based its approval on results from three trials in adults and children with diabetes, which showed a time to blood glucose recovery from severe hypoglycemia of 10 minutes following injection of 0.6 milligrams/0.6 milliliters of Zegalogue. 

FDA Grants Accelerated Approval to Large B-Cell Lymphoma Antibody

FDA recently granted accelerated approval to Zynlonta, a CD19-directed antibody and alkylating agent conjugate for adult patients with relapsed or refractory large B-cell lymphoma.

The agency based its approval on an open-label trial, LOTIS-2, which discovered that patients who received the antibody experienced an overall response rate of 48.3 percent, with a complete response rate of 24.1 percent.