FDA Update: January 2019

Jan 22, 2019

Express Scripts Office of Clinical Evaluation and Policy tracks some recent updates to the drug pipeline.

New Pill Bottle

New Indication for Tecentriq

Tecentriq® (atezolizumab – Genentech) received FDA approval on Dec. 6, 2018, as initial treatment for metastatic non-squamous non-small cell lung cancer (NSCLC) that does not have EGFR or ALK genomic tumor aberrations. It will be used in a combination that also includes Genentech’s Avastin® (bevacizumab) and chemotherapy (chemo) with paclitaxel and carboplatin. Tecentriq is a programmed death receptor-ligand 1 (PD-L1)-blocking antibody that helps the body’s immune system attack cancer cells. For the new indication, it will be given as a 1,200mg intravenous (IV) infusion once every three weeks. For the first four to six treatment cycles, Tecentriq will be followed by Avastin and chemo, all on the same day. Once chemo ends, Tecentriq and Avastin therapy will continue once every three weeks until the cancer progresses or the drug side effects become too harmful for the patient. Updated prescribing information is available here.

Tolsura Approved for Systemic Fungal Infections

On Dec. 11, 2018, the FDA approved Mayne Pharma Group’s Tolsura™ (SUBA®-itraconazole) capsules. A new formulation of an existing antifungal drug, it is indicated to treat certain adults who have aspergillosis, blastomycosis or histoplasmosis; serious fungal infections that usually strike patients whose immune systems are undermined by other medical conditions. Tolsura is manufactured using a technique (SUBA) that improves its solubility and makes its effectiveness more predictable. It will be released in January 2019, as 65mg capsules with directions for doses between 130mg and 260mg per day. Doses of 130mg can be taken once daily; higher amounts should be divided into two separate doses per day. A boxed warning on its label cautions that Tolsura can cause or aggravate heart failure and that it should not be taken at the same time as numerous other drugs that can have serious interactions with it. Complete prescribing information for Tolsura is located here.

Herzuma, a Biosimilar to Herceptin, Approved

Herzuma® (trastuzumab-pkrb), jointly developed by Celltrion and Teva Pharmaceutical Industries, was approved by the FDA on Dec. 14, 2018. It is a biosimilar to Genentech’s Herceptin® (trastuzumab). Indicated as part of multi-drug regimens, it treats certain breast cancers, including metastatic cases, that have tested positive for an increased quantity of human epidermal growth factor receptor 2 (HER2) receptors on the surface of the tumor cells (HER2+). Herzuma also can be used alone for metastatic HER2+ breast cancer that has been treated previously with chemo. Herzuma’s launch date has not yet been determined. Mylan and Biocon’s Ogivri (trastuzumab-dkst), the first biosimilar to Herceptin, was FDA approved in December 2017, but it has not yet launched in the U.S. All trastuzumab products have boxed warnings that they may cause birth defects, heart failure, respiratory collapse or severe allergic reactions. Full prescribing information for Herzuma can be found here.

Motegrity Approved for Chronic Idiopathic Constipation

Shire won FDA approval on Dec. 14, 2018, for Motegrity (prucalopride). It is the first selective serotonin-4 (5-HT4) receptor agonist approved to treat adults who have chronic idiopathic constipation (CIC). It will be taken orally once a day. In five clinical trials, approximately twice as many patients taking Motegrity regained normal bowel movement patterns as those taking a placebo tablet (19% to 38% for Motegrity versus 10% to 20% for placebo). Although no direct relationship has been established, some trial participants had suicidal thoughts or exhibited suicidal tendencies. Patients beginning therapy should report uncharacteristic mood changes to their healthcare providers. Shire has not yet announced its exact launch or pricing plans. Prescribing information is available here.

Long-Acting Asparagine, Asparlas, Approved

Asparlas (calaspargase pegol-mknl), an orphan-designated new version of an asparagine-specific enzyme, was approved by the FDA on Dec. 20, 2018. It is indicated for use in combination with chemo to treat acute lymphoblastic leukemia (ALL) for patients between the ages of one month and 21 years. A modified, longer-acting, form of previously available products, it works by decreasing the amounts of an amino acid, asparagine, in the blood. Normal cells can produce asparagine, but ALL cells cannot, so they disintegrate. Asparlas will be administered as an IV infusion once every three weeks as opposed to currently available forms that need to be given more often. Recommended dosing is 2,500 units/m2 of body surface area. It will be dispensed in single-dose vials containing 3,750 units of active drug. The manufacturer, Servier Pharmaceuticals, has not yet released its plans for pricing. Launch is expected in the first quarter of 2019. For prescribing information, please see here.

Pediatric Indication for Nplate

Under a priority review, the FDA approved Nplate® (romiplostim) on Dec. 14, 2018, to treat chronic immune thrombocytopenic purpura (ITP) for children as young as one year of age. Previously limited to use by adult patients, Nplate works similarly to thrombopoietin, a natural human protein that stimulates platelet production in the bone marrow. Chronic ITP is a rare autoimmune bleeding disorder characterized by low platelet count. It affects an estimated 4,000 U.S. patients who are younger than 18 years of age. If left untreated, ITP can result in anemia, internal bleeding and serious infections. Nplate is given as a once-weekly subcutaneous (SC) injection at one mcg/kg of body weight. An associated Risk Evaluation and Mitigation Strategy (REMS) and Medication Guide for patients outline possible severe side effects of using Nplate, which is available only through a restricted distribution program called Nplate NEXUS (Network of Experts Understanding and Supporting Nplate and Patients). Only healthcare providers and patients registered with the program are able to prescribe and receive Nplate. Revised prescribing information is located here.

Lynparza Receives New Indication

Lynparza® (olaparib) tablets were FDA approved on Dec. 19, 2018, as maintenance treatment for adult patients who have deleterious or suspected deleterious germline or somatic BRCA-mutated (gBRCAm or sBRCAm) advanced epithelial ovarian, fallopian tube or primary peritoneal cancer and who are in complete or partial response to first-line platinum-based chemo, as detected by an FDA-approved companion diagnostic test. Lynparza is a poly ADP-ribose polymerase (PARP) inhibitor that prevents cancer cells from repairing themselves after being damaged by chemo. It has prior indications for treating other types of ovarian cancer and for metastatic HER2-negative breast cancer that has mutations in the BRCA gene. The recommended dose is 300mg twice daily. Lynparza is marketed jointly by AstraZeneca and Merck. Its complete prescribing information can be found here.

Keytruda Approved for Merkel Cell Carcinoma

On Dec. 19, 2018, Merck’s Keytruda® (pembrolizumab) received accelerated approval from the FDA for the treatment of patients who have recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). Keytruda is a PD-1 blocker that helps the body’s immune system attack cancer cells. Originally approved on Sep. 4, 2014, to treat patients with unresectable or metastatic melanoma, Keytruda now has numerous indications for treating various kinds of cancer. For MCC, it will be administered once every three weeks as a 30-minute IV infusion. The recommended dose for treating children who have MCC is 2mg/kg up to a maximum of 200mg. For adults, the dose is 200mg. Treatments will be given for 24 months, until the cancer worsens or until the patient can no longer tolerate the drug. Continued approval of Keytruda for MCC depends upon confirmatory trials that demonstrate a clinical benefit. Full prescribing information is available here.

First Generic Launched for Canasa

Mylan announced on Dec. 17, 2018, that it has launched mesalamine rectal suppositories, 1,000mg, the company’s generic for Allergan’s Canasa®. Mesalamine suppositories are used topically to treat mild-to-moderate proctitis (inflammation of the rectal lining), which affects about one-third of patients who have an inflammatory bowel disease, such as ulcerative colitis. It is used once a day at bedtime for between three weeks and six weeks to relieve inflammation. As the first company to file for a generic, Mylan has 180-day exclusivity. IQVIA estimates that Canasa’s American sales amounted to $263 million in the 12 months that ended on Oct. 31, 2018.

Ultomiris Approved for Paroxysmal Nocturnal Hemoglobinuria

On Dec. 21, 2018, the FDA approved Alexion’s Ultomiris (ravulizumab-cwvz) injection for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that causes the destruction of red blood cells (hemolysis). Ultomiris is a long-acting C5 complement inhibitor that prevents hemolysis. It is administered every eight weeks by IV infusion; the dose depends on the patient’s weight. Alexion’s Soliris® (eculizumab), a complement C5 inhibitor, has been on the market since 2007 for the treatment of PNH. It is also approved for atypical hemolytic uremic syndrome (aHUS) and generalized myasthenia gravis (gMG). After induction, Soliris is administered as an IV infusion every two weeks, compared to every eight weeks for Ultomiris. The labeling for Ultomiris contains a Boxed Warning, highlighting an increased risk of serious meningococcal infections. Patents should be vaccinated with a meningococcal vaccine at least two weeks prior to receiving the first dose of Ultomiris. Patients should also be monitored for early signs of meningococcal infections. Ultomiris is available only through a restricted program under a REMS. Prescribers must enroll in the program and patients are required to receive a Medication Guide that describes the risks associated with the use of Ultomiris. Ultomiris is an orphan drug that was approved under FDA’s Priority Review pathway. Its launch date and pricing information are not known at this time. Full prescribing information can be found here.

Elzonris Approved to Treat Blastic Plasmacytoid Dendritic Cell Neoplasm

The FDA approved Stemline Therapeutics’ Elzonris (tagraxofusp-erzs) injection on Dec. 21, 2018, to treat patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). Often presenting as leukemia or evolving into acute leukemia, BPDCN is a rare and aggressive disease that can result in tumor cells in the blood, bone marrow, lymph nodes or skin. It more commonly occurs in patients 60 years of age and older with approximately 75% of cases occurring in men. Elzonris is a targeted therapy that selectively binds to the IL-3 receptor (CD123), which is overexpressed on multiple hematological cancer cells. Binding to this receptor allows the targeted delivery of the cytotoxin (diphtheria toxin) to the CD123-expressing cells, resulting in cell death. Following premedication, the recommended dose of Elzonris is 12mcg/kg of body weight, administered IV over 15 minutes, once daily on days one to five of a 21-day cycle. Availability and distribution plans are not known at this time. Labeling for Elzonris contains a Boxed Warning about the increased risk of capillary leak syndrome, which may be life-threatening or fatal if not properly managed. Elzonris was approved under the FDA’s Breakthrough Therapy and Priority Review programs. It also is an orphan drug. Complete prescribing information is available here.

Inbrija Approved for Parkinson’s Disease

Acorda Therapeutics received approval from the FDA on Dec, 21, 2018, for Inbrija (levodopa inhalation powder). A new inhaled formulation of levodopa, it is indicated for the intermittent treatment of OFF episodes for patients who have mild-to-moderate Parkinson’s disease that is being treated with carbidopa/levodopa. After levodopa has been used as long-term Parkinson’s treatment, its effectiveness may vary. Many patients experience OFF episodes -- breakthrough symptoms such as tremors and muscle stiffness despite continual treatment. The recommended dose is 84mg (two capsules) inhaled through the Inbrija inhaler device when needed, but no more than five times daily. Capsules should not be swallowed. Patients who also have a lung condition, such as asthma, or who have taken a nonselective monoamine oxidase (MAO) inhibitor, such as phenelzine, within the prior two weeks should not use it. Acorda plans to launch Inbrija during the first quarter of 2019 through a specialty pharmacy network. It will be supplied in boxes containing one inhaler and either 60 or 92 capsules that are individually blister packed. Full prescribing information is located here.

Elidel Generic Introduced

Pimecrolimus cream, 1%, a generic for Elidel® (Novartis), was released on the U.S. market by Teva Pharmaceutical Industries on Dec. 27, 2018. A topical immunomodulator, it is indicated for the short-term treatment of eczema (atopic dermatitis) for patients who are two years old or older, who are not immunocompromised and who have tried at least one other topical treatment that hasn’t worked for them. It is applied to affected areas of skin twice a day. After six weeks, treatment should be evaluated for effectiveness. Annual U.S. sales for Elidel amounted to over $218 million according to IQVIA.

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