FDA Update: December 2017

Dec 5, 2017
Express Scripts Office of Clinical Evaluation and Policy tracks some recent updates to the drug pipeline.

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Express Scripts’ Emerging Therapeutics team monitors actions from the U.S. Food and Drug Administration (FDA). Here are some important approvals and updates we tracked over the past month:

First Drug Approved to Treat Erdheim-Chester Disease

Based on results of a phase II basket study, the FDA approved Zelboraf® (vemurafenib - Genentech) tablets to treat Erdheim-Chester Disease (ECD) on Nov. 6, 2017. Basket studies enroll patients whose tumors have specific mutations rather than patients with cancers of specific organs. ECD is one in a group of related conditions that all involve overproduction of a specific type of white blood cells, which collect abnormally in bones, organs and other body tissues. It is believed to affect about 500 Americans. Most patients with it are diagnosed in their 50s or 60s, around three-quarters are men and about half have BRAF V600E mutations. Current treatments include corticosteroids, certain chemotherapy drugs, interferons, radiation and surgery. Zelboraf, a kinase inhibitor, also is indicated to treat patients who have inoperable or metastatic melanoma that has BRAF V600E mutations. Recommended dosing for both indications is 960mg (four tablets) taken at approximate 12-hour intervals. Prescribing information for Zelboraf is available here.

New Vaccine Approved to Prevent Hepatitis B

Heplisav-B™ [hepatitis B vaccine, recombinant (adjuvanted)], was approved by the FDA on Nov. 9, 2017, for preventing all subtypes of hepatitis B. It includes a Toll-like Receptor (TLR) 9 agonist that increases immune response to the hepatitis B surface antigen it contains. Indicated for adults, it will be given as two intramuscular (IM) injections spaced one month apart. Currently available hepatitis B vaccines require more doses to be administered over longer times. Additionally, Heplisav-B was effective for higher percentages of clinical trial participants receiving it than an already available vaccine was. Hepatitis B, a highly contagious, incurable viral infection of the liver, can be sexually transmitted or acquired from contact with blood infected with it. Untreated, it can result in cirrhosis, cancer or death. Because children now are vaccinated against hepatitis B, nearly all new diagnoses in the United States – about 3,400 acute cases and 14,500 chronic cases in 2015 – are among adults. The U.S. Centers for Disease Control and Prevention (CDC) recommends hepatitis B vaccinations for adults who have diabetes and those who may be at higher risk through their lifestyles, occupations or travel. Dynavax, Heplisav-B’s manufacturer, expects to launch it in the first quarter of 2018. Its full prescribing information is available here.

Pediatric Indication for Sprycel

A new indication was FDA approved on Nov. 10, 2017, for Bristol-Myers Squibb’s kinase inhibitor Sprycel® (dasatinib) tablets. Its new approval is for treating children and adolescents who are in the chronic phase of Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) and who weigh at least 10Kg (22 pounds). Only about 3% of children with leukemia have CML, but it often progresses more rapidly for young patients. Daily dosing of Sprycel for pediatric patients in clinical trials was 60mg/square meter of body surface area up to a total of 100mg per day. Doses should be evaluated once every three months and adjusted according to changes in the patient’s weight. Bone growth also should be watched since Sprycel has caused growth delays for some patients. Other major side effects may include blood abnormalities, excessive bleeding, heart irregularities and pulmonary hypertension. Sprycel also has adult indications to treat some types of CML and acute lymphoblastic leukemia (ALL) that are Ph+. It is supplied as 20mg, 50mg, 70mg, 80mg, 100mg and 140mg oral tablets. Revised prescribing information can be found here.

Fasenra Approved for Severe Asthma

The FDA approved Fasenra™ (benralizumab – AstraZeneca) on Nov. 14, 2017. An adjunct therapy, it will be used for maintenance treatment of patients at least 12 years old who have severe eosinophilic asthma. It works by blocking interleukin-5 (IL-5) receptors on the surfaces of eosinophils, a type of white blood cell. In clinical studies, patients using Fasenra along with their standard asthma drugs had improved lung function and fewer asthma attacks than patients receiving their usual asthma medications plus a placebo. Recommended dosing is 30mg given by a healthcare provider subcutaneously (SC) once every two months after the first three doses are administered at one-month intervals. AstraZeneca plans to launch Fasenra in the next few weeks at a first-year wholesale acquisition cost (WAC) of $38,000. Prescribing information can be found here.

FDA Approves the First Medication that Contains a Tracking Device

On Nov. 14, 2017, Otsuka received FDA approval for the first drug product that includes a sensor to track ingestion. Developed in co-operation with Proteus Digital Health, the drug, Abilify Mycite® (aripiprazole tablets with sensor), is an atypical antipsychotic with several indications that include some aspects of bipolar I disorder, major depression and schizophrenia. Each tablet contains an ingestible event marker (IEM) about the size of a sand grain. When the sensor comes in contact with stomach acid, a signal goes to a patch the patient wears. Information is transmitted to a smartphone app and the patient can designate healthcare providers and/or caregivers to receive the data. If the patient wishes, other information, such as activity levels, mood and sleep can be tracked, but those functions are not yet FDA approved. The initial launch for Abilify Mycite will be very limited – through a small group of providers and for carefully selected patients, only. Estimated cost is not available. Dosing recommendations are from 2mg to 15mg per day depending on the condition being treated, but no more than 30mg should be taken per day. Labeling for Abilify Mycite carries a boxed warning that the chance of death is increased for elderly patients who have dementia-related psychoses. Complete prescribing information can be found here.

Mepsevii Approved for Mucopolysaccharidosis VII

The FDA approved Mepsevii™ (vestronidase alfa-vjbk) injection on Nov. 15, 2017. It replaces an enzyme, beta-glucuronidase, which is missing for patients who have a very rare inherited condition known as mucopolysaccharidosis type VII (MPS VII). Indicated for both children and adults, it is given once every two weeks as a four-hour intravenous (IV) infusion at 4mg/Kg. Pricing information has not yet been announced. A boxed warning cautions that some patients have experienced severe allergic reactions from Mepsevii, so infusions should be given in a facility prepared to handle emergencies. Patients should remain at the place of administration for at least one hour after each infusion. Complete prescribing information for Mepsevii is available here.

Hemlibra Approved for Hemophilia A

On Nov. 16, 2017, the FDA approved Genentech’s Hemlibra® (emicizumab-kxwh) for routine prophylaxis 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. The recommended dose is 3 mg/kg injected SC once weekly for the first 4 weeks, followed by 1.5 mg/kg once weekly. Complete prescribing information is available here.

First Two-Drug Combination Approved to Treat HIV

Juluca®, ViiV Healthcare’s fixed-dose combination tablet, which contains 50mg of dolutegravir and 25mg of rilpivirine, was approved by the FDA on Nov. 21, 2017. Dolutegravir, branded as Tivicay® (ViiV) is a human immunodeficiency virus type 1 (HIV-1) integrase strand transfer inhibitor (INSTI) and rilpivirine, brand-name Edurant® (Janssen), is a HIV-1 non-nucleoside reverse transcriptase inhibitor (NNRTI). The combination is indicated for once daily oral dosing, with food, to treat adults who have HIV-1 infection that has been virologically suppressed to HIV-1 RNA levels of less than 50 copies per mL on a stable antiretroviral regimen for at least six months. Juluca replaces current therapy for patients with no history of treatment failure and no known substitutions associated with resistance to the individual components of the combination. Juluca’s complete prescribing information can be found here.

Sublocade Approved for Opioid Use Disorder

On Nov. 30, 2017, the FDA approved Indivior’s Sublocade™ (buprenorphine extended release), a once-monthly C-III injection for the treatment of moderate-to-severe opioid use disorder (OUD). It can be used by patients who have initiated treatment with a transmucosal buprenorphine-containing product, followed by dose adjustments for a minimum of seven days. Sublocade should be used as part of a complete treatment program that includes counseling and psychosocial support. The drug is injected SC in the abdominal region by a health care professional. The recommended dose is 300mg monthly for the first two months, followed by 100mg monthly maintenance doses. The maintenance dose can be increased to 300mg monthly if the benefits are determined to outweigh the risks. Indivior plans on launching Sublocade 100mg/5mL and 300mg/1.5mL syringes during the first quarter of 2018. Sublocade must be prescribed and dispensed as part of a Risk Evaluation and Mitigation Strategy (REMS) to ensure that the product is not distributed directly to patients. Complete prescribing information is available here.

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