18 FDA-Approved “Path-Breaking” Drugs of 2017
Only a few days are left to start the new year celebrations. Till now, the US FDA has approved over 40 drugs to treat a wide range of diseases, but some of them created more excitement than other. Here are the 18 innovative new drugs or therapies approved in 2017 that offer a pioneering or path-breaking way for disease treatment.
Disease: Metastatic Merkel cell carcinoma (mMCC)- a rare skin cancer
Active Ingredient: Avelumab
Date of Approval: 23-03-2017
First and the only FDA-approved treatment to treat mMCC patients.
mMCC is a rare and aggressive skin cancer. Less than 50 percent of the patients survive for more than a year, and less than 20 percent survive beyond five years. Bavencio targets PD-1/PD-L1 pathway (proteins found on the body’s immune cells and some cancer cells) and rejuvenates the body’s immune system to target cancer cells by blocking these interactions. The drug significantly improved the overall response rate and duration of response in patients.
Disease: Moderate-to-Severe eczema (atopic dermatitis)
Active Ingredient: Dupilumab
Date of Approval: 28-03-2017
First and the only FDA-approved biologic medicine to treat moderate-to-severe atopic dermatitis.
Atopic dermatitis is a chronic inflammatory skin disease generally referred as eczema. It is twice a month injection used with or without topical corticosteroids. Dupixent acts like a targeted immune system therapy that binds to two important proteins or inflammatory compounds known as IL (interleukin)-4 (IL-4) and IL-13, thus inhibiting the inflammatory response which plays a major role in developing atopic dermatitis.
Disease: Moderate-to-Severe Plaque Psoriasis
Active Ingredient: Guselkumab
Date of Approval: 17-07-2017
First and the only biologic drug approved that selectively blocks IL-23.
Tremfya showed comparable results with the AbbVie’s blockbuster anti-inflammatory drug, Humira®. Psoriasis is a chronic, autoimmune inflammatory disorder, resulting due to the overproduction of skin cells that causes scaly, red lesions, itching, discomfort, and pain. Around 125 million people suffer from this condition across the globe. Tremfya selectively targets and blocks IL-23 which plays play a key role in the development of immune-mediated inflammatory diseases. Patients treated with Tremfya experienced significantly greater improvements in skin clearance and other measures of disease activity compared with Humira.
Disease: Carcinoid Syndrome Diarrhea
Active Ingredient: Telotristat ethyl
Date of Approval: 28-02-2017
First and the only oral treatment to treat carcinoid syndrome diarrhea.
Xermelo is approved to treat carcinoid syndrome diarrhea in cancer patients with advanced neuroendocrine tumors. Carcinoid syndrome is a rare and debilitating disease, affecting people with advanced neuroendocrine tumors. Xermelo targets the overproduction of serotonin inside neuroendocrine tumors.
Disease: Batten Disease- hereditary disorder affecting children
Active Ingredient: Cerliponase alfa
Date of Approval: 27-04-2017
First FDA-approved therapy to treat a specific form of Batten disease.
Batten disease is a fatal, inherited disorder of the nervous system that mostly begins in childhood characterized by the sudden development of seizures, behavioral changes, progressive dementia, visual loss, loss of the ability to walk and talk and cerebral atrophy in kids. Brineura is an enzyme replacement therapy to treat gradual loss of walking ability in pediatric patients three years of age and older with a specific form of Batten disease- Late-Infantile CLN2 disease, also known as tripeptidyl peptidase-1 (TPP1) deficiency.
The active ingredient- cerliponase alfa of the therapy is a recombinant form of human TPP1, the enzyme deficient in patients with CLN2 disease. It is administered into the cerebrospinal fluid of the patients by infusion under a sterile environment.
Disease: Acute Myeloid Leukemia (AML)- a form of blood cancer
Active Ingredient: Enasidenib
Date of Approval: 01-08-2017
First and the only FDA-approved therapy to treat AML patients with an IDH2 mutation.
About 8 to 19 percent of patients with AML have a mutation in IDH2 gene. IDH2 mutation is a rare genetic mutation. Idhifa is an oral, targeted inhibitor of the IDH2 enzyme. The IDH2 enzyme plays a vital role in the metabolism and energy production of cells, helping in cell growth. Mutation in IDH2 enzyme blocks normal blood cell development, causing an abundance of immature blood cells. Idhifa works by blocking several enzymes that promote proliferation of cell.
Disease: B-cell acute lymphoblastic leukemia (ALL)– a rare hard-to-treat pediatric blood cancer
Active Ingredient: tisagenlecleucel-T
Date of Approval: 30-08-2017
First Ever CAR-T cell-based gene therapy approved by FDA.
Kymriah is a personalized cancer therapy developed from patient’s own T cells. In the therapy, patient’s own T-cells are taken out and genetically modified to include a new gene containing a specific protein- chimeric antigen receptor (CAR). The CAR directs the T-cells to identify and kill the tumor cells having a specific antigen (CD19) on their surface. After the modification, the cells are infused back into the patients to kill cancer cells.
Disease: Acute Myeloid Leukemia
Active Ingredient: Midostaurin
Date of Approval: 28-04-2017
First and the only targeted treatment for newly diagnosed FLT3-mutated AML.
Rydapt plus chemotherapy treat AML patients, having a genetic mutation in FLT3 gene. FLT3-mutated AML is a rare and aggressive type of cancer that develops in blood and bone marrow and worsens quickly, if not treated early. FLT3 mutation has been observed in about 30 to 35 percent of AML patients. It is one of the most commonly mutated genes in AML. Rydapt is a kinase inhibitor and works by blocking several enzymes that promote cell growth.
Disease: Diffuse large B-cell lymphoma (DLBCL)- an aggressive form of blood cancer
Active Ingredient: axicabtagene ciloleucel
Date of Approval: 18-10-2017
First CAR T-cell based gene therapy to treat DLBCL in adults.
Yescarta is a customized gene therapy developed from patient’s own T-cells to combat cancer cells. DLBCL is the most common type of non-Hodgkin lymphoma (NHL)- a form of blood cancer in adults. NHL arises in certain cells of immune system and can spread rapidly in the body. CAR T-cells specifically bind to the CD19 proteins found on the cancer tumor cells and delete them from the body.
Disease: Multiple Sclerosis
Active Ingredient: Ocrelizumab
Date of Approval: 28-03-2017
First and only FDA-approved treatment for severe forms of multiple sclerosis.
It is the first treatment for both relapsing and primary progressive forms of multiple sclerosis. Multiple sclerosis is an auto-immune disease where an individual’s immune system attack’s one’s own healthy tissue. It generally affects the brain and spinal cord. Ocrelizumab is a humanized anti-CD20 monoclonal antibody which acts on CD20 marker on B lymphocytes. B lymphocytes express a protein, CD20 on the surface. Ocrevus is designed to specifically bind to these B-cells to suppress the immune activity in the body.
Disease: Giant cell arteritis, Cytokine Release Syndrome
Active Ingredient: Tocilizumab
Date of Approval: 22-05-2017
First FDA-Approved Drug to specifically treat giant cell arteritis (GCA) and CAR-T cell therapy-induced cytokine syndrome.
GCA is a chronic and severe autoimmune disease that causes inflammation in the lining of the arteries. It can result in stroke or blindness if left untreated. Cytokine release syndrome induced by CAR T-cell therapy is a common and potentially dangerous complication of anti-T cell injections. It is a fatal inflammatory response of the body against the therapy. Actemra is an IL-6 receptor antagonist which binds to IL-6 receptors and hinders its inflammatory response.
Company: Chemo Research
Disease: Chagas Disease- a parasitic infection
Active Ingredient: Benznidazole
Date of Approval: 29-08-2017
First FDA-Approved Drug to treat Chagas disease.
Chagas disease is caused by the infection of the parasite, Trypanosoma cruzi which can be transmitted through various routes like blood transfusion. It primarily affects the people living in the rural parts of Latin America. It is approved by FDA to treat children with Chagas disease between age group 2 to 12 years old.
Company: Symbiomix Therapeutics
Disease: Bacterial vaginosis
Active Ingredient: Secnidazole
Date of Approval: 18-09-2017
First-of-its-kind treatment for bacterial vaginosis (BV) in adult women.
BV is the most common gynecologic infection in the U.S., affecting 21 million women annually. If left untreated, it can increase the risk of sexually transmitted diseases, including gonorrhea, herpes, trichomoniasis, and HIV, and can also increase the risk of premature delivery. Solosec™ is the first and the only single-dose oral antibiotic to treat BV in adult women. Solesec is a next-generation, 5-nitroimidazole antibiotic which is highly effective and well-tolerated in a single dose.
Company: Neurocrine Biosciences
Disease: Tardive Dyskinesia
Active Ingredient: Valbenazine
Date of Approval: 11-04-2017
First FDA-approved drug to treat tardive dyskinesia.
Tardive dyskinesia is a neurological disorder characterized by repetitive and uncontrolled muscular movements. Ingressa is a vesicular monoamine transporter 2 (VMAT2) inhibitor. The exact mechanism of the drug is unknown. The drug is given orally.
Disease: Tardive dyskinesia and Huntington’s disease associated Chorea
Active Ingredient: deutetrabenazine
Date of Approval: 30-08-2017
First and only FDA-approved product to treat both tardive dyskinesia in adults and chorea associated with Huntington’s disease.
Tardive dyskinesia is characterized by repetitive and uncontrolled movements of face, tongue, lips and other body parts. Detetrabenazine is a deuterated form of the vesicular monoamine transporter 2 inhibitor tetrabenazine (Xenazine, Lundbeck Inc). It is an oral drug which was first approved in April 2017 to reduce chorea (abnormal uncontrolled movement disorder) in patients with Huntington’s disease.
Company: Adamas Pharmaceuticals
Disease: Dyskinesia in Parkinson’s Disease patients
Active Ingredient: amantadine
Date of Approval: 24-08-2017
First FDA-approved medication to treat dyskinesia in Parkinson’s disease patients.
In the patients, dyskinesia is caused due to levodopa-based Parkinson’s disease treatment that is characterized by involuntary body movements. Gocovri is a high dose 274mg amantadine extended-release capsules, taken once-daily that consistently supplies high levels of amantadine throughout the day when dyskinesia occurs.
Disease: Erdheim-Chester Disease- a rare blood cancer
Active Ingredient: Vemurafenib
Date of Approval: 06-11-2017
First treatment for adult patients with Erdheim-Chester Disease (ECD) having BRAF V600 genetic mutation.
Nearly, 54 percent patients with ECD have the BRAF V600 mutation. It is a slow-growing cancer of the blood, originating in the bone marrow. Zelboraf is a selective inhibitor of the activated BRAF V600 gene. BRAF V600 gene encodes for the proteins required for the proliferation of the cancer cells. Therefore, Zelboraf hampers the growth and development of the cancer cells.
Company: Ultragenyx Pharmaceutical
Disease: Mucopolysaccharidosis type VII (MPS VII)- a rare, genetic disorder
Active Ingredient: Vestronidase alfa-vjbk
Date of Approval: 15-11-2017
First FDA approved treatment for pediatric and adult patients with MPS VII.
MPS VII is a lysosomal storage disorder that occurs due to deficiency of an enzyme called beta-glucuronidase. The enzyme causes an abnormal buildup of toxic materials in the body’s cells. This affects the tissues and organs of the body. Mepsevii is an enzyme replacement therapy that replaces the missing enzyme in the body. MPS VII is a very rare and progressive condition that occurs in less than 150 patients worldwide.
Featured Image Credit: Bigstockphoto (new drugs 2017)