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New Drug Approvals in 2017: Advancing Innovation

Medically reviewed on Dec 31, 2017 by L. Anderson, PharmD.

HCV Agents for All Six Genotypes

Over time, hepatitis C virus (HCV) can lead to liver scarring, cirrhosis, liver cancer and death. Health experts recommended that all "baby boomers" - those born from 1946 to 1964 - be tested for HCV to determine treatment need. Additional novel, life-saving oral HCV meds that allow bypass of interferon and ribavirin have been approved since the introduction of Harvoni and Sovaldi several years ago. The year 2017 was no exception.

  • Mavyret (glecaprevir and pibrentasvir) from AbbVie: Mavyret is the first FDA-approved 8-week treatment for all chronic HCV genotypes (1-6) in adult patients without cirrhosis (liver disease) and without previous treatments. Standard treatment length for HCV was previously 12 weeks or longer. Mavyret can also be used in patients with compensated cirrhosis or previously treated with other HCV agents. In studies, 92% to 100% of patients had no virus detected in the blood 12 weeks after finishing treatment, suggesting a cure. Common side effects include headache and fatigue.

  • Vosevi (sofosbuvir, velpatasvir and voxilaprevir) from Gilead: Vosevi is approved by the FDA for retreatment of adults with chronic HCV of two types: either genotype 1, 2, 3, 4, 5, or 6 previously treated with an NS5A inhibitor regimen, or genotype 1a or 4 previously treated with a sofosbuvir regimen without an NS5A inhibitor. Vosevi, given once daily, is used in patients without cirrhosis or with compensated cirrhosis who failed other HCV treatments. In studies, 96% to 97% of patients who received Vosevi had no virus detected 12 weeks after finishing treatment. Common adverse reactions are headache, fatigue, diarrhea and nausea.

Learn More: Oral Hepatitis C Treatments - The Evolving Landscape

First Time Approvals for Rare Indications

Over 600 drugs and biologic products for rare diseases have been approved by the FDA since 1983. Orphan drugs are specifically developed to treat a rare medical condition, affecting fewer than 200,000 people nationwide. Several were approved in 2017:

  • Mepsevii (vestronidase alfa) from Ultragenyx Pharmaceuticals: A recombinant human lysosomal beta glucuronidase approved for treatment of Mucopolysaccharidosis Type VII (MPS VII, Sly syndrome). MPS VII is an inherited, rare genetic condition with various skeletal and organ abnormalities, and possible intellectual disability. Learn More: Mepsevii Approval History

  • Zelboraf (vemurafenib) from Genentech: The first drug approved for a rare blood cancer, Erdheim-Chester Disease. Zelboraf is a kinase inhibitor that works by blocking certain enzymes that promote cell growth. Zelboraf was first approved in 2011 to treat metastatic melanoma. Learn More: Zelboraf Approval History

  • Brineura (cerliponase alfa) from BioMarin Pharmaceutical: Approved for late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a form of Batten disease, a rare inherited disorder that primarily affects the nervous system. Learn More: Brineura Approval History

  • Xermelo (telotristat ethyl) from Lexicon: Approved for the treatment of carcinoid syndrome diarrhea in cancer patients with metastatic neuroendocrine tumors (mNET). Xermelo is an oral tryptophan hydroxylase inhibitor used in combination with somatostatin analog (SSA) therapy. Learn More: Xermelo Approval History

Ocrevus: A Novel Treatment Among Multiple Sclerosis Drugs

Ocrevus (ocrelizumab): the much anticipated multiple sclerosis (MS) treatment from Genentech was given the green light from the FDA in March 2017.

  • Ocrevus is the first approved medicine for primary progressive forms of multiple sclerosis.
  • It is a humanized monoclonal antibody designed to selectively target CD20-positive B cells, a first-in-class B cell targeted therapy.
  • Ocrevus is also the first treatment approved for both primary progressive multiple sclerosis (PPMS) and relapsing forms of MS.

In these forms of MS, Phase III studies demonstrated that:

  • Ocrevus slowed disability progression and reduce signs of disease activity in the brain (MRI lesions).
  • In the RMS group, relapses per year were reduced by nearly one-half.
  • Mild to moderate side effects include infusion reactions and upper respiratory tract infections.

Two New VMAT2 Inhibitors for Movement Disorders

In addition to Xenazine (tetrabenazine) approved in 2008, two more vesicular monoamine transporter 2 inhibitors (VMAT2 inhibitors) were approved in 2017.

VMAT2 inhibitors are used to treat the involuntary movements (chorea) of inherited Huntington’s disease, or the unwanted movements of the tongue, face, mouth or other body parts of tardive dyskinesia, usually due to use of antipsychotic medications.

How do VMAT2 inhibitors work? The VMAT2 inhibitors block the VMAT2 protein leading to a lower amount of neurotransmitter (chemical messengers like dopamine and serotonin) between nerves (synaptic cleft) for uptake which reduces the involuntary movements.

VMAT2 inibitors approved in 2017:

  • Ingrezza (valbenazine) from Neurocrine Biosciences: The first drug for the treatment of tardive dyskinesia, FDA-approved in April 2017.

  • Austedo (deutetrabenazine) also approved April 2017 from Teva Pharmaceuticals: This medication is the first approved to treat both tardive dyskinesia in adults and chorea associated with Huntington's disease.

Innovation: First In Class Agents

New medications that are first-in-class are often breakthrough therapies for previously untreatable conditions:

  1. Vyzulta (latanoprostene bunod) ophthalmic solution, 0.024% from Valeant Pharmaceuticals: A nitric oxide donating prostaglandin receptor agonist that is used to lower intraocular (eye) pressure in patients with open-angle glaucoma or ocular hypertension. See the FDA-approval history for Vyzulta.
  2. IDHIFA (enasidenib) from Celgene: a first-in-class, oral, targeted inhibitor of mutant isocitrate dehydrogenase 2 (IDH2) for the treatment of patients with relapsed or refractory acute myeloid leukemia (AML) with an IDH2 mutation. See the FDA-approval history for Idhifa.
  3. Hemlibra (emicizumab-kxwh) from Genentech: a first-in-class bispecific factor IXa- and factor X-directed antibody approved to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A who have developed antibodies called Factor VIII inhibitors. See the FDA-approval history for Hemlibra.

Expanding Gene Therapies

Immunotherapy made history in 2017 with the approval of three gene therapies for patients with serious or rare conditions and limited treatment options.

CAR-T Cell Therapy for Cancer

Kymriah (tisagenlecleucel or CTL019) from Novartis: In August 2017, the FDA approved the first cell-based gene therapy, Kymriah. Kymriah is a genetically-modified T-cell immunotherapy customized for each patient using their own T-cells, a type of white blood. It is used in patients up to 25 years of age with advanced B-cell acute lymphoblastic leukemia (ALL) not responsive to other treatments or in relapse.

Yescarta (axicabtagene ciloleucel) from Gilead/Kite Pharma: Given FDA clearance in October 2017, Yescarta was the first CAR T-Cell therapy approved for certain adults with non-Hodgkin (NHL) lymphoma after failing at least two other kinds of treatment. CAR-T cell therapy can be linked with serious side effects, such as cytokine release syndrome (CRS), which can be fatal.

Eye Disease

Luxturna (voretigene neparvovec-rzyl) from Spark Therapeutics: Luxturna was approved in Dec. 2017 as the first directly administered gene therapy that targets mutations in a specific gene. Luxturna is used in patients with biallelic RPE65-mediated inherited retinal disease (IRD), a form of vision loss that can lead to blindness. It is an adeno-associated viral (AAV) vector gene therapy that delivers a normal copy of the RPEE65 gene directly to retinal cells.

Shingrix: Now First Line for Shingles

In October 2017, the FDA cleared GSK's Shingrix (zoster vaccine recombinant, adjuvanted) vaccine for the prevention of herpes zoster (shingles) in adults 50 years and older. Shingles is caused by the reactivation of the varicella zoster virus (VZV), the same virus that causes chickenpox, and often strikes older or immunocompromised adults.

  • Shingrix is a non-live, recombinant subunit vaccine given as two intramuscular injections, with the second dose given 2 to 6 months after the first.
  • In studies of more than 38,000 people spanning a follow-up period of 4 years, Shingrix was effective against shingles in greater than 90% of all age groups. Zostavax is effective in roughly 50% of patients
  • The incidence of postherpetic neuralgia (PHN), a form of chronic nerve pain and the most common complication linked with shingles was also reduced.

The big impact from this vaccine comes from it's boosted effectiveness compared to Zostavax. The CDC’s Advisory Committee on Immunization Practices (ACIP) now recommends the Shingrix vaccine over Zoster Vaccine Live (Zostavax). Plus, those who received Zostavax in the past should now also receive Shingrix, which equates to over 20 million people.

Breast Cancer Breakthroughs: New CDK 4/6 Inhibitors

In addition to Pfizer's CDK4/6 inhibitor Ibrance (palbociclib) approved 2015, Kisqali and Verzenio, also CDK4/6 inhibitors, were approved in 2017 to target metastatic breast cancer -- advanced breast cancer that has spread in the body.

Kisqali (ribociclib) from Novartis:

  • Kisqali is recommended for use with an aromatase inhibitor for postmenopausal women with HR+/HER2- metastatic breast cancer.
  • In studies, patients who received Kisqali plus letrozole reduced their risk of disease worsening or death by 44% over letrozole alone, and over half saw their tumors shrink by at least 30%.
  • Common side effects include low white blood cells, upset stomach, hair loss, and fatigue, among others.
  • CYP450 3A4 enzyme drug interactions and monitoring for heart arrhythmias figure prominently in the drug label.

Verzenio (abemaciclib) from Lilly:

  • Verzenio is approved for use in 2 patient populations: either with fulvestrant (Faslodex) in patients with HR-positive, HER2-negative advanced or metastatic breast cancer that progressed after endocrine therapy; or as monotherapy for patients who previously received endocrine therapy and chemotherapy.
  • In studies used with fulvestrant, the median progression-free survival (length of time tumors did not grow after treatment ) was 16.4 months compared to 9.3 months for the fulvestrant/placebo group. In stand-alone studies, 19.7% of patients taking Verzenio experienced complete or partial shrinkage of their tumors for a median of 8.6 months.
  • Common side effects include diarrhea, low levels of certain white blood cells (neutropenia and leukopenia), nausea, vomiting, infections, and fatigue, among others.

Learn More: Breast Cancer Therapy: Right On Target

New 2017 Biosimilars

The approval pace was quickened in 2017 when it came to biosimilars. A biosimilar is a biological product that is similar to a reference biologic product and for which there are no clinically meaningful differences in terms of safety, purity, and potency.

A biosimilar is not considered a “generic” in the same way as a traditional drug, but, like generics, cost savings for the healthcare system and the consumer are expected to be significant. According to the RAND Corporation, biosimilars could save the U.S. health system close to $44 billion in the next decade.

Biosimilars approved in 2017 include:

  • Renflexis (infliximab-abda) from Samsung Bioepis, approved in April 2017 and Ixifi (infliximab-qbtx) from Pfizer, approved December 2017 for various inflammatory diseases such as rheumatoid arthritis, Crohn's disease, and plaque psoriasis; both biosimilar to Remicade
  • Cyltezo (adalimumab-adbm) from Boehringer Ingelheim, approved in August 2017 for inflammatory diseases such as rheumatoid arthritis, ankylosing spondylitis, and plaque psoriasis; a biosimilar to Humira.
  • Mvasi (bevacizumab-awwb) from Amgen, approved in September 2017 for non-small cell lung cancer, colorectal cancer, glioblastoma multiforme, renal cell carcinoma, and cervical cancer; a biosimilar to Avastin and the first biosimilar for a cancer indication.
  • Ogivri (trastuzumab-dkst) from Mylan GmbH, approved in December 2017 for breast cancer and stomach cancer, a biosimilar to Herceptin.

Learn More: What Are Biosimilars? Top Facts You May Not Know

Immune Checkpoint Inhibitors in 2017

Bavencio (avelumab):

  • Bavencio is the first FDA-approved treatment for metastatic Merkel Cell Carcinoma (MCC) and is used in patients 12 years and older. It is also approved for advanced urothelial carcinoma.
  • MCC is rare form of skin cancer, and urothelial carcinoma are cancers that occur most commonly in the bladder, but also the ureters and renal pelvis.
  • A programmed death ligand-1 (PD-L1) blocking antibody from EMD Serono, Bavencio received accelerated FDA-approval in March 2017 for MCC and in May 2017 for urothelial carcinoma.

Imfinzi (durvalumab):

  • Imfinzi (durvalumab), from AstraZeneca, also an anti-PD-L1 blocker, was cleared in May 2017 for the treatment of patients with locally advanced or metastatic urothelial carcinoma.
  • Imfinzi was also approved under the FDA’s accelerated approval pathway, based on tumor response rate and durability of response.
  • In studies, Imfinzi had an objective response rate (ORR) of 17%, regardless of PD-L1 status, with an ORR of 26.3% in patients with PD-L1 high-expressing tumors. Among 31 patients who responded to treatment, 14 patients (45%) had ongoing responses of at least 6 months and 5 patients (16%) at least 12 months. Of all evaluable patients, 2.7% achieved complete response.

Other approved Immune Checkpoint inhibitors include: Keytruda (pembrolizumab) and Opdivo (nivolumab) approved in 2014, and Tecentriq (atezolizumab) approved in 2016

Learn More: Immune Checkpoint Inhibitors: Boosting the Cancer Battle

New Interleukin Inhibitors in 2017

Dupixent (dupilumab) from Regeneron:

  • In 2017, the FDA cleared Dupixent, the first targeted biologic therapy for adults with moderate-to-severe atopic dermatitis, an advanced form of eczema. Dupixent is classified as an interleukin-4 receptor alpha antagonist. Review the Dupixent approval history.

Siliq (brodalumab) from Valeant Pharmaceuticals:

  • Siliq is now approved for adults with moderate-to-severe plaque psoriasis. Siliq is an anti-interleukin-17-receptor monoclonal antibody. Learn more about Siliq from its pivotal clinical trials.

Tremfya (guselkumab) from Janssen:

Kevzara (sarilumab) from Regeneron/Sanofi:

  • Kevzara is an interleukin-6 receptor (IL-6R) antagonist approved for moderate-to-severe rheumatoid arthritis (RA) in patients who have a poor response to disease-modifying antirheumatic drugs (DMARDs). Kevzara may be used alone or in combination with methotrexate (MTX) or other DMARDs. See the pivotal MOBILITY studies that compared Kevzara plus MTX versus placebo plus MTX for RA.

Juluca: A Novel 2-Drug Regimen for HIV

In November 2017 the FDA approved ViiV Healthcare’s Juluca (dolutegravir and rilpivirine), a single-tablet, fixed-dose, two-drug regimen of the approved drugs dolutegravir (Tivicay) and rilpivirine (Edurant).

  • Juluca is the first complete treatment regimen containing only two drugs, instead of three or more drugs, for HIV maintenance.
  • It is used in certain adults with suppressed HIV-1 virus on a stable medication regimen for at least 6 months, with no history of treatment failure and no resistance to the individual components of Juluca.
  • In clinical trials, Juluca was effective in keeping the virus suppressed and comparable to those who continued their current anti-HIV drugs.
  • The most common side effects in patients taking Juluca were diarrhea and headache.

Learn More: HIV & AIDS Update: New Treatments, Easier Options

Zejula: New PARP Inhibitor for Ovarian Cancer

In March 2017, the FDA approved Zejula (niraparib) for the maintenance treatment of women with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer following response to platinum-based chemotherapy.

Zejula is an oral, once-daily poly(ADP-ribose) polymerase (PARP) inhibitor, and the first PARP inhibitor that does not require BRCA mutation or other biomarker testing. Zejula, from Tesaro, joins other PARP inhibitors Rubraca (rucaparib) approved in 2016 from Clovis Oncology, and Lynparza (olaparib) approved in 2014 from AstraZeneca.

In pivotal clinical Zejula studies in patients with a germline BRCA mutation, the median progression-free survival (PFS) was 21 months, compared to 5.5 months for the control group. In the group without a BRCA mutation, median PFS was 9.3 months compared with 3.9 months in the placebo group.

Ozempic: A Once-Weekly GLP-1 Receptor Agonist

The FDA approved Novo Nordisk’s Ozempic (semaglutide), a glucagon-like peptide-1 (GLP-1) analog administered once-weekly for the treatment of adults with type 2 diabetes in December 2017. Ozempic is suitable for once-weekly dosing due to high albumin binding and a longer half-life. Ozempic is approved for use in two therapeutic doses, 0.5 mg and 1 mg, and will be launched in the Ozempic pre-filled pen.

In Phase 3 studies, Ozempic showed significant reductions in A1c compared with placebo, sitagliptin (Januvia) and exenatide extended-release (Bydureon); a reduction in body weight was also seen. The most common adverse reactions reported in 5% or greater of patients treated with Ozempic were: nausea, vomiting, diarrhea, abdominal pain and constipation.

Other GLP-1 receptor agonists, also known as incretin mimetics, include:

New Kinase Inhibitors in 2017

Rydapt (midostaurin) from Novartis:

Alunbrig (brigatinib) from Takeda Pharmaceuticals:

Nerlynx (neratinib) from Puma Biotechnology:

  • In July 2017, the FDA OK'd Nerlynx (neratinib), a once-daily oral tyrosine kinase inhibitor for the extended treatment of early stage HER2-positive breast cancer, following adjuvant trastuzumab (Herceptin) therapy. Nerlynx is the first extended adjuvant therapy to further lower this type of breast cancer recurrence. Review pivotal clinical trial results and Nerlynx side effects.

Calquence (acalabrutinib) from AstraZeneca:

Aliqopa: A New PI3K Inhibitor

In September 2017 the FDA cleared Bayer's Aliqopa (copanlisib) for relapsed follicular lymphoma, a slow-growing type of B-cell non-Hodgkin lymphoma, a cancer of the lymph system.

  • Aliqopa is a phosphatidylinositol-3-kinase (PI3K) inhibitor that works by blocking several enzymes that promote cell growth.
  • In a single-arm study with 104 patients, 59% of patients had a complete or partial response for a median 12.2 months.
  • Common side effects of Aliqopa include high blood sugar levels, diarrhea, decreased energy, and high blood pressure, among others.

Zydelig (idelalisib) from Gilead is also a PI3K inhibitor approved in 2014 for the treatment of relapsed chronic lymphocytic leukemia, follicular B-cell non-Hodgkin lymphoma, and small lymphocytic lymphoma.

Vabomere: For Complicated Urinary Tract Infections

In August 2017, the FDA approved Vabomere (meropenem and vaborbactam) for adults with complicated urinary tract infections (UTIs), including pyelonephritis caused by specific bacteria.

  • Vabomere contains meropenem, an antibacterial, and vaborbactam, a new β-lactamase inhibitor which inhibits certain types of resistance mechanisms used by bacteria.
  • In studies with 545 adults with complicated UTI's, including those with pyelonephritis, roughly 98% of patients treated with Vabomere had a cure or improvement in symptoms and a negative urine culture test.
  • The most common side effects in patients taking Vabomere were headache, infusion site reactions and diarrhea, among others.

More Breakthroughs: Onwards to 2018

There is no doubt that 2017 ushered in some important medical advances -- consider these important research discoveries:

  • The advent of direct gene therapy and treatments tailored for an individual patient such as CAR-T cell therapy is a historical breakthrough that is set to change the future of cancer.
  • Continued progress for rare diseases and approval of orphan drug products provide advances for patients with life-threatening diseases and limited treatment options.
  • New antibacterial agents can be inserted into treatment algorithms where antibacterial resistance and therapeutic failures are commonplace but serious.
  • Oral cures for hepatitis C virus that target all six genotypes, allowing patients to bypass difficult side effects with older treatments.
  • Bacterial and viral vaccines innovations that prevent disease, especially important for vulnerable populations such as children and the elderly.

But what might happen in 2018? The FDA will be busy reviewing studies for more new, innovative medications, monitoring safety for drugs already on the market, and pushing forward to find advances for diseases that afflict public health, such as type 2 diabetes, Alzheimer's disease, and life-threatening cancers.

It's exciting. Stay up-to-date with new approvals in 2018 by following the Weekly Drug News Round-Up.

Finished: New Drug Approvals in 2017: Advancing Innovation

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