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New FDA Drug Approvals in 2019 by Indication: Part 2

First Report Managed Care's December issue includes a guide to 2019 FDA Drug Approvals by indication. Find Part 1 here


BRUKINSA (zanubrutinib)
Manufacturer: BeiGene USA Inc

Approval Date: November 14, 2019

FDA approved Brukinsa capsules for treatment for patients with mantle cell lymphoma (MCL)who have received at least one prior treatment.

The FDA approval of Brukinsa is based on evidence from two trials that showed about 84% of patients had a complete or partial shrinkage of their tumors after treatment. In Trial 1, all patients with MCL who had at least one prior therapy received two Brukinsa capsules twice daily until their disease progressed or experienced unacceptable side effects. Results were measured by tumor shrinkage calculated with positron emission tomography. 

Trial 2 enrolled patients with B-cell lymphoma, including MCL patients who were previously treated with Brukinsa. Trial 2 patients received either two Brukinsa tablets twice daily or three capsules once daily until disease progression or they presented unacceptable side effects. 

Brukinsa comes in capsule form and is taken either as two capsules twice daily or four capsules once daily. Trial 2 patients had tumor shrinkage assessed using computed tomography or magnetric resonance imaging. 

The most common side effects of Brukinsa are low blood cell counts, upper respiratory tract infections, rash, bruising, diarrhea and cough.

Brukinsa was approved under FDA’s accelerated approval program. More trials are ongoing to assess whether there is a clinical benefit.

INREBIC (fedratinib)
Manufacturer: Celgene Corporation

Approval Date: August 16, 2019

FDA approved Inrebic capsules for treatment in adult patients with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis. Myelofibrosis is a chronic disorder where scar tissue forms in the bone marrow and the production of the blood cells moves from the bone marrow to the spleen and liver, causing organ enlargement.

The FDA approval of Inrebic is based on evidence from a clinical trial of 192 patients with myelofibrosis conducted in Asia, Australia, Brazil, Canada, Europe, Mexico, South Africa, and the United States. All patients had spleens measured at the beginning of the trial using either magnetic resonance imaging or computed tomography scan. Then patients received either Inrebic or a placebo once daily for 6 months. Neither patients nor health care providers had knowledge of which patients received treatment vs placebo. Spleens were measured again at the end of the trial period. 

For patients receiving Inrebic (n=96), 37% saw a decreased spleen measurement of 35% or more. Placebo patients (n=96) saw a 1% reduction. 

The most serious adverse effect reported was a fatal brain malfunction called Wernicke encephalopathy, most commonly found in patients with a vitamin B1 deficiency. Other serious side effects could include low red blood cells, low platelet count, vomiting, diarrhea, and abnormal liver and pancreatic tests. The most common side effects are diarrhea, nasusea, anemia, and vomiting. 

Inrebic is a capsule taken orally once daily. Health care professionals are advised to assess thiamine levels in all patients prior to starting Inrebic, during treatment and as clinically indicated. If encephalopathy is suspected, Inrebic should be immediately discontinued.

ROZLYTREK (entrectinib)
Manufacturer: Genentech, Inc.

Approval Date: August 15, 2019

FDA approved Rozlytrek, to treat adult patients with metastatic nonsmall cell lung cancer (NSCLC) whose tumors are ROS1-positive and to treat adult and pediatric patients aged 12 years and older with solid tumors. 

The FDA approval of Rozlytrek for patients with metastatic NSCLC and for patients with solid tumors was based on evidence from four clinical trials, Trial 1 (EudraCT 2012-000148-88), Trial 2 (NCT02097810), Trial 3 (NCT02568267), and Trial 4 (NCT02650401).

Forty (78%) of 51 patients with NSCLC who received Rozlytrek experienced complete or partial shrinkage of their tumors. Tumor shrinkage lasted more than 12 months for 55% % of those patients. Fifty-seven percent of 54 patients with various solid tumors who received Rozlytrek experienced complete or partial shrinkage of their tumors which lasted for more than 12 months for 45% of them. The most common cancer locations were the lung, salivary gland, breast, thyroid, and colon/rectum.

Rozlytrek can cause serious side effects including congestive heart failure, nervous system problems, bone fractures, liver toxicity, increased uric acid in the blood, heart rhythm problems, and vision problems. The most common side effects are tiredness, constipation, taste changes, body swelling, dizziness, and diarrhea.

Rozlytrek is a capsule taken orally once daily for patients with a biomarker no younger than 12 years. 

TURALIO (pexidartinib)
Manufacturer: Daiichi Sankyo

Approval Date: August 2, 2019

FDA approved Turalio tablets for treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not responsive to improvement with surgery.

The FDA approval of Turalio is based on results from a multicenter, international clinical trial comprising 120 patients, 59 of whom received a placebo. After 25 weeks of treatment, 38% of patients who received Turalio saw improvement in overall response rate compared to 0% of the placebo group. 

The complete response rate was 15% and the partial response rate was 23%. A total of 22 of 23 responders who had been followed for a minimum of 6 months following the initial response maintained their response for 6 or more months, and a total of 13 of 13 responders who had been followed for a minimum of 12 months following the initial response maintained their response for 12 or more months.

The most adverse response to Turalio included risk of serious to potentially fatal liver injury, so health care professionals should closely monitor liver function and test throughout treatment. This reason is also why Turalio is only available through the Risk Evaluation and Mitigation Strategy program.

Most common side effects include abnormal liver tests, hair color changes, and tiredness. 

Turalio is a capsule taken twice daily on an empty stomach.

NUBEQA (darolutamide)
Manufacturer: Bayer

Approval Date: July 30, 2019

FDA approved Nubeqa, an androgen receptor inhibitor, for the treatment of patients with nonmetastatic castration-resistant prostate cancer. 

The FDA approval is based on results from the phase 3, ARAMIS trial that evaluated Nubeqa plus androgen deprivation therapy which demonstrated a highly significant improvement in the primary efficacy endpoint of metastasis-free survival, with a median of 40.4 months vs 18.4 months for placebo plus androgen deprivation therapy (P<.0001).

In the ARAMIS trial, both arms showed a 9% discontinuation rate due to adverse reactions. The most frequent adverse reactions requiring discontinuation in patients who received Nubeqa included cardiac failure (0.4%) and death (0.4%).  Adverse reactions occurring more frequently in the Nubeqa arm (≥2% over placebo) were fatigue (16% vs 11%), pain in extremity (6% vs 3%) and rash (3% vs 1%). Nubeqa was not studied in women and there is a warning and precaution for embryo-fetal toxicity.

The most common side effects experienced are fatigue, extremity pain, and rash. 

Patients take Nubeqa as two tablets orally, twice daily with food.  

XPOVIO (selinexor)
Manufacturer: Karyopharm Therapeutics Inc.

Approval Date: July 3, 2019

FDA approved Xpovio, a nuclear export inhibitor, in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody.

The FDA approval of Xpovio is based on evidence from a clinical trial (NCT02336815) of 202 patients with multiple myeloma whose disease came back after, or did not respond to, previous treatments. The trial was conducted at 58 sites in United States and Europe.

The 202 participants received Xpovio in combination with dexamethasone twice a week until their disease progressed or side effects became toxic. In the trial, 21 of 83 patients (25%) treated with Xpovio in combination with dexamethasone experienced improvement in their disease that lasted about 4 months.

The most common side effects of Xpovio are low blood counts, nausea, fatigue, decreased appetite, decreased weight, diarrhea, vomiting, low blood sodium, constipation, shortness of breath, and upper respiratory tract infections.

Xpovio is administered as 4 tablets (80 mg each) taken orally in combination with dexamethasone on days 1 and 3 of each week. Dosage may be decreased if the patient cannot tolerate the side effects. 

POLIVY (polatuzumab vedotin-piiq)
Manufacturer: Genentech, Inc

Approval Date: June 10, 2019

FDA approved Polivy, a novel antibody-drug conjugate, to treat adult patients with diffuse large B-cell lymphoma that has progressed or returned after at least two prior therapies, to be used in combination with the chemotherapy bendamustine and a rituximab product (a combination known as BR). 

The FDA approval of Polivy is based on evidence from one clinical trial where 40 patients received either Polivy in combination with BR or BR only every 21 days for six treatment cycles. Both patients and health care providers knew which treatment had been given. Efficacy was evaluated by measuring how many patients showed remission in either group and how long the remission lasted.

Results showed that 63% of patients assigned to Polivy plus BR had complete or partial shrinkage of their rumors and 40% complete shrinkage. 

In comparison, of 40 patients assigned to BR alone, 25% had a remission, and 18% had a complete remission after finishing treatment.

Potential serious side effects of Polivy may include peripheral neuropathy, infusion related allergic reactions, bone marrow suppression, infections including progressive multifocal leukoencephalopathy, tumor lysis syndrome, liver damage, and harm to an unborn baby.

Polivy’s most common side effects include low blood cell counts, nerve damage, fatigue, diarrhea, fever, decreased appetite, and harm to an unborn baby. 

Polivy is given by a health care provider directly into the bloodstream as an intravenous infusion every 21 days for 6 cycles together with BR.

PIQRAY (alpelisib)
Manufacturer: Novartis Pharmaceuticals Corporation

Approval Date: May 24, 2019

FDA approved Piqray tablets to be used in combination with the FDA-approved endocrine therapy fulvestrant, to treat postmenopausal women, and men, with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CA-mutated, advanced or metastatic breast cancer (as detected by an FDA-approved test) following progression on or after an endocrine-based regimen.

The FDA approval of Piqray tablets is based on efficacy data from the SOLAR-1 trial, a randomized trial comprising 572 postmenopausal women and men with HR-positive, HER2-negative, advanced or metastatic breast cancer whose cancer had progressed while on or after receiving an aromatase inhibitor. 

Results from the trial showed the addition of Piqray to fulvestrant significantly prolonged progression- free survival (median of 11 months vs. 5.7 months) in patients whose tumors had a PIK3CA mutation.

Common side effects of Piqray are high blood sugar levels, increase in creatinine, diarrhea, rash, decrease in lymphocyte count in the blood, elevated liver enzymes, nausea, fatigue, low red blood cell count, increase in lipase, decreased appetite, stomatitis, vomiting, weight loss, low calcium levels, prolonged activated partial thromboplastin time, and hair loss.

Piqray is taken as two tablets once daily with food in combination with fulvestrant.

BALVERSA (erdafitinib)
Manufacturer: Janssen Products, LP

Approval Date: April 12, 2019

FDA approved Balversa, a treatment for adult patients with locally advanced or metastatic bladder cancer that has a type of susceptible genetic alteration known as FGFR3 or FGFR2, and that has progressed during or following prior platinum-containing chemotherapy. 

The FDA approval of Balversa is based on evidence from a single clinical trial conducted in Asia, Europe, and the United States. All 87 patients received Balversa once daily as a tablet taken orally until either their disease worsened or they developed an unacceptable side effect. Thirty-two percent of 87 patients who received Balversa experienced complete or partial shrinkage of their tumors.

Balversa was approved under FDA’s accelerated approval program. Patients should be selected for therapy with Balversa using an FDA-approved companion diagnostic device.

Common side effects of taking Balversa include increased phosphate level, mouth sores, feeling tired, change in kidney function, diarrhea, dry mouth, nails separating from the bed or poor nail formation, change in liver function, low sodium levels, decreased appetite, change in sense of taste, anemia, dry skin, dry eyes, and hair loss. 

Balversa is a tablet taken once daily for a total dose of 8 mg. During treatment, the Balversa dose may be increased to 9 mg if needed.



BEOVU (brolucizumab–dbll)
Manufacturer: Novartis Pharmaceuticals Corporation

Approval Date: October 7, 2019

FDA approved Beovu for the treatment of wet age-related macular degeneration. It is the first approval in its class to offer both greater fluid resolution versus aflibercept and the ability to maintain eligible wet AMD patients on a 3-month dosing interval immediately after a 3-month loading phase with uncompromised efficacy.

The FDA approval of Beovu is supported by evidence from the phase 3 HAWK and HARRIER clinical trials, in which it demonstrated noninferiority vs aflibercept in mean change in best-corrected visual acuity at year one (week 48). 

In both clinical trials, approximately 30% of patients gained at least 15 letters at year one. In HAWK and HARRIER, Beovu showed greater reduction in central subfield thickness as early as week 16 and at year one, and fewer patients had intra-retinal and/or sub-retinal fluid. 

In two head-to-head clinical trials, patients on Beovu (brolucizumab) achieved vision gains that were non-inferior to aflibercept at year one with longer treatment intervals in a majority of patients.

The most common adverse events exhibited during Beovu’s trials were blurred vision, cataract, conjunctival hemorrhage, vitreous floaters, and eye pain. 

Beovu is an injection, administered by a health care professional directly inside the eyeball every month for 3 months, and then again every 8 to 12 weeks.




TRIKAFTA (elexacaftor/ivacaftor/tezacaftor))
Manufacturer: Vertex Pharmaceuticals

Approval Date: October 21, 2019

FDA approved Trikafta, the first triple combination therapy available to treat patients with the most common cystic fibrosis mutation, for patients aged 12 years and older with cystic fibrosis who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

The FDA approval of Trikafta is supported by data from two trials. The first trial was a 24-week, randomized, double-blind, placebo-controlled trial in 403 patients who had an F508del mutation and a mutation on the second allele that results in either no CFTR protein or a CFTR protein that is not responsive to ivacaftor or tezacaftor/ivacaftor alone. The second trial was a 4-week, randomized, double-blind, active-controlled trial in 107 patients who had two identical F508del mutations. 

In both trials, the benefit of Trikafta was assessed by change in percent predicted forced expiratory volume (ppFEV1). In Trial 1 Trikafta was compared to placebo after 4 weeks of treatment and in Trial 2 to active comparator after 4 weeks of treatment.

Trikafta improved lung function by allowing air to move easier. In patients treated with Trikafta ppFEV1 increased.

Serious side effects of Trikafta could include increase liver enzymes and clouding of lens in the eye. Most commonly reported side effects include headache, upper respiratory infections, abdominal pain, diarrhea, rash and elevated liver enzymes.

Trikafta treatment comes in co-packaged orange and blue tablets. Two orange tablets (containing a combination of three drugs elexacaftor, tezacaftor and ivacaftor) are taken orally in the morning. One light blue tablet (containing ivacaftor) is taken orally in the evening.

Manufacturer: Mylan Laboratories Limited

Approval Date: August 14, 2019

FDA approved Pretomanid tablets in combination with bedaquiline and linezolid for the treatment of a specific type of highly treatment-resistant tuberculosis (TB) of the lungs.

The FDA approval of Pretomanid tablets is supported by a trial, comprising 109 patients aged 17 to 60 years, conducted in South Africa, that showed that 95 out of 107 patients (89%) had no TB bacteria in the sputum 6 months after completing the treatment.

Pretomanid is approved under FDA’s Limited Population Pathway for Antibacterial and Antifungal Drugs, which provides approval of antibacterial and antifungal drugs to treat serious or life-threatening infections in a limited population of patients with unmet need.

The most common adverse effects included peripheral neuropathy, acne, anemia, nausea, vomiting, headache, increased liver enzymes (transaminases and gamma-glutamyltransferase), indigestion (dyspepsia), rash, increased pancreatic enzymes (hyperamylasemia), visual impairment, low blood sugar, and diarrhea.

Pretomanid is a tablet taken orally once daily for 26 weeks. It must be used in combination with two other antimicrobial drugs (bedaquiline and linezolid).



VYLEESI (bremelanotide)
Manufacturer: AMAG Pharmaceuticals, Inc

Approval Date: June 21, 2019

FDA approved Vyleesi, a drug to activate melanocortin receptors, to treat acquired, generalized hypoactive sexual desire disorder (HSDD) in premenopausal women. HSDD is acquired and generalized if the woman has not had problems with low sexual desire in the past, and if she has symptoms no matter the type of sexual activity, the situation, or the sexual partner.

The approval of Vyleesi is supported by evidence from two 24-week, randomized, double-blind, placebo-controlled trials in 1247 premenopausal women with acquired, generalized HSDD. 

Participants in the trial used Vyleesi 2 or 3 times per month and no more than once a week. 

In these trials, about 25% of patients treated with Vyleesi had an increase of 1.2 or more in their sexual desire score (scored on a range of 1.2 to 6.0, with higher scores indicating greater sexual desire) compared to about 17% of those who took placebo. There was no difference between treatment groups in the change from the start of the study to end of the study in the number of satisfying sexual events. Vyleesi does not enhance sexual performance.

The most common side effect of Vyleesi are nausea and vomiting, flushing, injection site reactions and headache. More serious side effects include a temporary increase in blood pressure, decrease in heart rate, and darkening of the gums or skin on face and breasts. 

Vyleesi is administered using an autoinjector subcutaneously in the abdomen or thigh, 45 minutes before sexual activity. 

EVENITY (romosozumab-aqqg)
Manufacturer: Amgen, Inc

Approval Date: April 9, 2019

FDA approved Evenity, a monoclonal antibody that blocks the effects of the protein sclerostin and works mainly by increasing new bone formation, for the treatment of osteoporosis in postmenopausal women at high risk of breaking a bone. It is designed for those with a history or osteoporotic fracture or multiple risk factors for fracture, or those who have failed or are intolerant to other osteoporosis therapies.

The FDA approval of Evenity is supported by two clinical trials involving over 11,000 women with postmenopausal osteoporosis. The first trial showed a 73% lower risk of fracture for patients receiving the Evenity injections vs placebo. This benefit was maintained over the second year of the trial when Evenity was followed by 1 year of denosumab compared to placebo followed by denosumab. In the second trial, 1 year of treatment with Evenity followed by 1 year of alendronate reduced the risk of a new vertebral fracture by 50% compared to 2 years of alendronate alone. Evenity followed by alendronate also reduced the risk of fractures in other bones (nonvertebral fractures) compared to alendronate alone.

Evenity increased the risk of cardiovascular death, heart attack, and stroke in the alendronate trial but not in the placebo trial. Therefore, patients with history of heart attack or stroke should not use Evenity. 

Common side effects include joint paint and headache, as well as reactions at the injection site. 

One dose of Evenity consists of two injections, one immediately following the other, given once a month by a health care professional. The bone forming effect of Evenity wanes after 12 doses so more than 12 doses should not be used. If osteoporosis therapy is needed after the 12 doses, patients should begin an osteoporosis treatment that reduces bone breakdown.

ZULRESSO (brexanolone)
Manufacturer: Sage Therapeutics, Inc.

Approval Date: March 19, 2019

FDA approved the Zulresso injection for intravenous (IV) use for the treatment of postpartum depression (PPD) in adult women.

In two trials, patients with moderate or severe PPD who were given Zulresso, achieved more improvement of depressive symptoms than patients given placebo.

The efficacy of Zulresso was shown in the two clinical studies in participants who received a 60-hour continuous IV infusion of Zulresso or placebo and were then followed for 4 weeks. One study included patients with severe PPD and the other included patients with moderate PPD. The primary endpoint in the study was the mean change from baseline in depressive symptoms as measured by a depression rating scale. In both placebo-controlled studies, Zulresso demonstrated superiority to placebo in improvement of depressive symptoms at the end of the first infusion. The improvement in depression was also observed at the end of the 30-day follow-up period.

The most common side effects of Zulresso were sleepiness, dry mouth, loss of consciousness, and hot flashes.

Zulresso will be available only through the Zulresso Risk Evaluation and Mitigation Strategies (REMS) Program that requires the drug be administered by a health care provider in a certified health care facility. The REMS program requires that patients be enrolled in the program prior to administration of the drug. 

Zulresso is given as an IV infusion (injected into a vein) by a healthcare provider. The IV infusion takes about 60 hours (2½ days) to complete. The dose depends on the patient’s weight.

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