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2016 New Drug Approvals: The Year That Was

Medically reviewed on Dec 26, 2016 by L. Anderson, PharmD.

2016: Drug Approvals Slowed to a Crawl

No matter what you think about the year 2016, one thing is for certain: many of us are glad it’s over. Although the political scene was a fast-paced frenzy, the FDA slowed down novel drug approvals to a crawl in the year past.

Novel therapeutic agents -- innovative products that meet an unmet medical need or advance public health care in a significant way -- continue to hit the market, and 2016 was no exception. However, the FDA approved only 22 novel drugs in 2016, shrinking from a record approval of 45 novel drugs in 2015 and 41 drugs in 2014. Here’s our quick review of the top new approvals of 2016, including many novel drugs, and a sneak peek of possibilities on tap for 2017.

More Hepatitis C Breakthroughs

Hepatitis C virus (HCV) affects over 3 million Americans and can lead to liver impairment, cirrhosis or even liver failure. HCV treatments continue to make headway in covering multiple genotypes in 2016.

  • Zepatier (elbasvir and grazoprevir) from Merck, January 2016
  • Epclusa (sofosbuvir and velpatasvir) from Gilead, June 2016

Merck's once-daily oral HCV treatment Zepatier received approval soon after the 2016 New Year. Zepatier, a NS5A replication complex inhibitor and NS3/4A protease inhibitor combination is used with or without ribavirin in adults for HCV genotypes 1 and 4. In June FDA gave the go-ahead to Gilead’s Epclusa, an oral, once-daily nucleotide analog polymerase inhibitor and NS5A inhibitor used for all HCV genotypes 1 through 6 -- with or without cirrhosis.

Easier Treatments for HIV

Today's latest HIV treatments coupled with early diagnosis have boosted patient outcomes. In fact, most people with HIV who are treated early and receive ongoing treatment can live a near normal life span today. Significant 2016 HIV approvals include:

  • Odefsey (emtricitabine/rilpivirine/tenofovir alafenamide [TAF] from Gilead, March 2016
  • Descovy (emtricitabine and tenofovir alafenamide [TAF] from Gilead, April 2016

Odefsey is a 3-drug, fixed-dose non-nucleoside reverse transcriptase inhibitor (NNRTI) and nucleoside/tide reverse transcriptase inhibitor (NRTI) approved as a once-daily complete oral regimen for the treatment of HIV-1 infection. Odefsey is a new version of Complera that contains tenofovir alafenamide (TAF) instead of tenofovir disoproxil fumarate (TDF). Descovy is also a once-daily NRTI used with other medications for HIV treatment in patients 12 years and older who weigh at least 35 kg (77 lb).

Lilly’s Taltz Injection for Plaque Psoriasis

Psoriasis is a chronic autoimmune disease in which skin cells are overturned quickly. This continual replacement of skin leads to raised red plaques that can be flaky, itchy, and unsightly. In March, the FDA approved Eli Lilly's Taltz (ixekizumab), a humanized interleukin-17A antagonist, to treat adults with moderate-to-severe plaque psoriasis.

In Phase 3 studies of over 3,800 patients, 87 to 90 percent of patients treated with Taltz saw a significant improvement in their psoriasis by 3 months. Patients can learn to inject the drug using the autoinjector. Common side effects with Taltz include injection site reactions, upper respiratory tract infections, nausea, and fungal skin infections.

Biosimilars Surge Ahead

2016 might be called the year of the biosimilars, with a record three new agents winning FDA approval:

  • Inflectra (infliximab-dyyb) by Celltrion, April 2016
  • Erelzi (etanercept-szzs) from Sandoz, August 2016
  • Amjevita (adalimumab-atto) by Amgen, September 2016
These latest approvals are biosimilar to some well-known monoclonal antibody brand names: Inflectra is biosimilar to Remicade, Erelzi is biosimilar to Enbrel, and Amjevita is the first biosimilar to Humira. However, none of the new biosimilars can be automatically substituted at the pharmacy level; the physician must specify the name on the prescription. These agents are used to treat various autoimmune-type diseases, from rheumatoid arthritis to Crohn's disease. Analysts predict biosimilars in the U.S. will be sold at discounts of 10 to 40 percent from the branded competitors.

Parkinson’s Disease Psychosis: Nuplazid Approved

In April, the FDA approved Acadia’s Nuplazid (pimavanserin), the first atypical antipsychotic to treat hallucinations and delusions associated with Parkinson’s disease psychosis (PDP). Nuplazid shows no measurable activity at dopaminergic receptors, but instead exhibits inverse agonist and antagonist activity at serotonin receptors.

In clinical trials, Nuplazid was shown to be superior to placebo in decreasing psychosis without worsening motor symptoms. The recommended dose of Nuplazid is two-17 mg tablets taken once daily with or without food. Common side effects include swelling, nausea, and confusion.

Added Orphan Drugs Gain Headway

Roughly 15,000 new cases of rare chronic lymphocytic leukemia (CLL) are diagnosed each year; less than 1% of all cancers diagnosed. AbbVie's Venclexta (venetoclax), an oral B-cell lymphoma-2 (BCL-2) inhibitor that exhibits a 17p deletion, was approved in April. In studies, 106 patients took increasing oral doses of Venclexta once daily for 5 weeks and 80% either saw improvement or their cancer fully disappear. Common side effects include low red and white blood cell count, diarrhea, nausea, and respiratory infections.

Primary biliary cholangitis (PBC) is a rare autoimmune liver disease, often found in women over 40 years, that destroys bile ducts over time. However, treating PBC can allow for a normal life expectancy. In May, the FDA granted accelerated approval for Intercept’s Ocaliva (obeticholic acid), a first-in-class farnesoid X receptor (FXR) agonist for PBC. In clinical trials, Ocaliva met the primary endpoint in almost half of patients. Side effects commonly seen are itching, fatigue, stomach pain, and rash, among others.

Tecentriq for Two New Oncology Indications

Tecentriq (atezolizumab), the first available programmed death-ligand 1 (PD-L1) immune checkpoint inhibitor, was originally approved in May 2016 for bladder cancer then later in the year for non-small cell lung cancer (NSCLC).

Tecentriq is usually given by intravenous infusion over 60 minutes every 3 weeks. In studies, common side effects included fatigue, decreased appetite, nausea, urinary tract infection, fever, and constipation. Tecentriq also has the potential to cause infection and more serious, but less common, immune-mediated side effects that involve healthy organs, including the lungs, colon and endocrine system.

New Drugs for Rare Genetic Diseases

Duchenne Muscular Dystrophy is the most common childhood form of muscular dystrophy and is caused by a mutation in the dystrophin gene. The first medication for this condition, Exondys 51 (eteplirsen), a morpholino antisense oligomer, cleared the FDA in September and is given as an intravenous infusion once weekly. Exondys 51 was approved under the FDA's accelerated approval based on a surrogate endpoint likely to result in clinical benefit, but the FDA is requiring further studies from the manufacturer, Sarepta Therapeutics, to confirm.

Biogen's Spinraza (nusinersen), a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide, was approved at year's end as the first drug for spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle strength and movement. Spinraza is given as a spinal cord area injection. In an interim analysis of efficacy, 40% of patients treated with Spinraza reached motor milestones, but no control patients met these endpoints.

Lartruvo: Soft Tissue Sarcoma

The FDA granted accelerated approval to Lilly's Lartruvo (olaratumab) in October. Lartruvo is to be used with the chemotherapy agent doxorubicin for soft tissue sarcoma (STS) that cannot be cured with radiation or surgery. Lartruvo, a platelet-derived growth factor receptor alpha (PDGFR-α) blocking antibody to slow or stop tumor growth, is the first new initial treatment for soft tissue sarcoma in over 40 years.

In clinical trials, Lartruvo plus doxorubicin led to a significant improvement in median overall survival - 26.5 months compared to 14.7 months for patients who received doxorubicin alone. Common side effects included nausea, fatigue, low white blood cells (neutropenia), and muscle pain, among others.

Zinplava For C. Dif Prevention

Merck’s Zinplava (bezlotoxumab) was approved in October to help prevent a recurrence of Clostridium difficile infection (CDI) in high risk adults who are receiving antibacterial drug treatment for CDI. Symptoms of CDI can include mild-to-severe diarrhea, abdominal pain and fever.

Zinplava is a human monoclonal antibody that binds with C. difficile toxin B to prevent its effects on human cells. Common adverse reactions include nausea, fever and headache. Heart failure, a serious adverse reaction, was reported as well.

Lower Dose Tenofovir for Hepatitis B

Chronic hepatitis B virus (HBV) is a life-threatening liver virus that affects up to 2.2 million people in the U.S.

  • Vemlidy (tenofovir alafenamide fumarate, TAF) from Gilead, November 2016

Tenofovir alafenamide (TAF) was already included in 3 recently approved HIV combination treatments. Now Vemlidy is approved as a single agent for adults with chronic HBV infection with compensated liver disease. Vemlidy is a tenofovir prodrug with similar antiviral effectiveness and comes as a 25 milligram (mg) oral tablet taken once daily with food. Lab results show Vemlidy could be safer than conventional tenofovir due to lower dosing which might help protect the kidney and bones.

Rubraca for Ovarian Cancer

In an end-of-year, accelerated approval that came 2 months early, FDA cleared Clovis Oncology's Rubraca (rucaparib), a poly (ADP-ribose) polymerase (PARP) inhibitor indicated for the treatment of advanced mutant BRCA ovarian cancer. Rubraca is approved for women with advanced ovarian cancer who have been treated with two or more chemotherapies and whose tumors have a specific BRCA gene mutation.

In studies, 54% of patients who received Rubraca had complete or partial shrinkage of their tumors lasting a median of 9.2 months. Common side effects of Rubraca include nausea, fatigue, vomiting, low levels of red blood cells (anemia), and stomach pain, among others.

Looking Ahead: 2017

While the number of notable drugs approved from year-to-year may change, the FDA's job is never done. Here are some notable drugs in development that may be up for possible approval in 2017:

Need to look at other past approvals? You can access the New Drug Approvals Archive for a full historical overview back to 2002.

Finished: 2016 New Drug Approvals: The Year That Was

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