FDA Approved Drugs: July 2020

The Express Scripts Office of Clinical Evaluation and Policy lists the drugs that were approved by the Food and Drug Administration (FDA) in June 2020. (Estimated Reading Time: ~10 mins.)
FDA Update

Bynfezia Added to Drug File

Sun Pharmaceutical’s Bynfezia Pen (octreotide) injection, 2,500mcg/mL recently was added to the Express Scripts’ Drug File. Approved by the FDA in January 2020, it is an analogue of the hormone somatostatin that blocks growth hormone, glucagon, insulin and other body secretions. Given by subcutaneous (SC) injection, it is administered between two times and four times a day. At varying doses, it treats adults who have acromegaly, treats severe diarrhea and flushing associated with metastatic carcinoid tumors and also treats severe diarrhea due to vasoactive intestinal peptide tumors (VIPomas). Bynfezia Pens are dispensed in boxes of one or two multi-use devices each containing 2.8mL (7,000mcg) of octreotide. Although several other octreotide products, including generics, are available in the U.S., none is interchangeable with Bynfezia, which was approved as a branded product. Bynfezia will be added to Express Scripts’ specialty drug list. For its prescribing information, look here.

Nyvepria, Fourth Neulasta Biosimilar, Receives FDA Approval

Nyvepria (pegfilgrastim-apgf), Pfizer’s biosimilar to Neulasta® (pegfilgrastim - Amgen), was approved by the FDA on June 10, 2020. It is indicated to decrease the risk of infections for patients who receive cancer drugs that interfere with the bone marrow’s production of blood cells and that are associated with a clinically significant incidence of febrile neutropenia, an adverse effect of some chemo. Although multiple variables are involved in febrile neutropenia, an estimated 60,000 Americans being treated for cancer develop it, annually. Pegfilgrastim is a PEGylated form of the granulocyte colony-stimulating factor (G-CSF) analog filgrastim, giving it a longer duration of action. G-CSF causes cells in bone marrow to produce more neutrophils. For Nyvepria, the usual recommended dose is 6mg -- one single-dose prefilled syringe -- injected SC. Doses for patients who weigh less than 45kg (about 100 pounds) are based on the patient’s weight. Nyvepria is administered once during each chemo cycle, at least one day after chemo is completed and 14 days or more before the next treatment. In addition to the brand product, Nyvepria will compete with other biosimilars -- Fulphila® (pegfilgrastim-jmdb – Mylan/Biocon), Udenyca® (pegfilgrastim-cbqv – Coherus BioSciences) and Ziextenzo (pegfilgrastim-bmez – Sandoz), which already are available in the U.S. None of the biosimilars is interchangeable with Neulasta or with each other, however. Nyvepria will be added to Express Scripts’ specialty drug list. Pfizer expects a launch in the second half of 2020, but an exact date and pricing have not been announced. Prescribing information is here.

Opdivo Approved for New Indication

Bristol Myers Squibb’s programmed death receptor-1 (PD-1) inhibitor, Opdivo® (nivolumab), was approved for a new indication from the FDA on June 10, 2020. It now is indicated for the treatment of patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine and platinum-based chemo. After priority review, approval was based on results from a clinical trial that showed patients receiving Opdivo had greater overall survival (OS) -- 10.9 months vs 8.4 months for those using taxane chemo, such as docetaxel or paclitaxel, regardless of PD-L1 expression levels. A 240mg dose of Opdivo is infused intravenously (IV) every two weeks or a 480mg dose is infused every four weeks until cancer worsens or the patient can no longer tolerate using the drug. Opdivo is associated with several possible immune-related adverse effects in the lungs, intestines, kidneys, liver, skin or brain, which can be severe or life threatening. The FDA initially approved Opdivo in December 2014 as a Breakthrough Therapy for advanced melanoma. It also has several additional indications, either alone or in combination with other drugs, to treat various other types of cancer. Opdivo’s full prescribing information is found here.

Uplizna Approved for Neuromyelitis Optica Spectrum Disorder

On June 11, 2020, the FDA approved Viela Bio’s Uplizna (inebilizumab-cdon) for the treatment of adult patients who have neuromyelitis optica spectrum disorder (NMOSD) and who are anti-aquaporin-4 (AQP4) antibody positive. NMOSD is a rare autoimmune disease that attacks the optic nerve, spinal cord and brain stem. Affecting approximately 10,000 patients in the United States, it can cause blindness and paralysis. Patients also can lose bladder and bowel control, suffer nerve pain and experience respiratory failure. Approximately 80% of patients who have NMOSD have AQP4 antibodies. Approval of Uplizna was based on a clinical trial showing that 161 AQP4-positive patients who have NMOSD treated with Uplizna had a 77% reduction in the risk of an attack compared to placebo. In addition, 89% of patients remained relapse-free six months after treatment compared to 58% in the placebo group. Uplizna, a humanized CD19-directed monoclonal antibody, is administered as an IV infusion over 90 minutes. Patients should be pretreated with a corticosteroid, an antihistamine and an anti-fever medication to reduce the risk of infusion-related reactions. The recommended initial dose is 300mg followed two weeks later by a second 300mg infusion. Starting six months after the initial infusion, maintenance doses of 300mg should be given every six months. Pricing information is not yet available. Uplizna, which has Orphan Drug status and also is designated as a Breakthrough Therapy, will be added to the Express Scripts specialty drug list. For full prescribing information, look here.

FDA Approves Mylan/Biocon’s Insulin Glargine 

On June 11, 2020, the FDA approved Semglee® (insulin glargine – Mylan/Biocon) as a biologic product under the Biologic Price Competition and Innovation Act. A long-acting (basal) insulin, it is injected SC at the same time once every day to manage blood sugar levels for adults who have type 1 or type 2 diabetes and for children and teens who have type 1 diabetes. Dosing is individualized according to the patient’s blood sugar levels. As a new brand, Semglee is not automatically interchangeable with other insulin glargine products, Lantus® (Sanofi) or Basaglar® (Lilly/Boehringer Ingelheim), even though they have the same amino acid sequences. In addition to Lantus and Basaglar, Semglee will compete with other long-acting insulins, including Levemir® (insulin detemir – Novo Nordisk), Tresiba® (insulin degludec – Novo Nordisk) and Toujeo® (insulin glargine – Sanofi). It will be available in both 10mL multi-dose vials and 3mL disposable prefilled pens that contain 100 units of insulin/mL. A launch date and pricing information are yet to be determined. Here is its full prescribing information.

New Dosage Form and Indication for Tivicay

On June 12, 2020, the FDA approved ViiV Healthcare’s Tivicay PD (dolutegravir) tablets, 5mg, for oral suspension. An integrase strand transfer inhibitor (INSTI), it is used in combination with other drugs that treat HIV-1. Tivicay PD is given once daily for patients who are four weeks or older, who weigh 3kg (about 7 pounds) or more and who have not been given an INSTI previously. The tablets easily dissolve in liquids and doses are based on the child’s weight. At the same time, the FDA extended the pediatric indication for Tivicay film-coated tablets, 50mg, to children who weigh 20kg (about 45 pounds) or more. It originally was limited to patients at least six years old and at least 30kg. A date for the U.S. launch of Tivicay PD has not yet been announced. It will be dispensed in bottles of 60 tablets with a dose cup and an oral syringe. Prescribing information for both dosage forms is here.

Gardasil 9 Approved for Head and Neck Cancer

Under the FDA’s Accelerated Approval process, Merck’s Gardasil® 9 (human papillomavirus vaccine 9-valent, recombinant) was granted a new indication on June 12, 2020. It now is approved to prevent oropharyngeal (mouth and throat) and head or neck cancers that result from infections with specific strains of human papillomavirus (HPV). The U.S. Centers for Disease Control and Prevention (CDC) estimates that approximately 14 million Americans are infected with HPV each year. Many individuals are not even aware of the infections and most cases clear up without treatment. Some HPV strains may cause genital warts and some patients who have HPV may develop cancers of the anus, cervix, penis, throat or vagina. Originally FDA approved in 2014 to prevent infections for patients between nine years and 26 years of age, Gardasil 9 provides protection against nine strains of HPV. It does not treat any HPV infection the patient already has, however; and it offers no protection against HPV strains the patient was exposed to before being vaccinated. In 2018 its use was expanded to adults between 27 years and 45 years of age. It is given in a series of two or three intramuscular (IM) injections over six or 12 months, depending on the patient’s age. Here is prescribing information.

Zepzelca approved for Small Cell Lung Cancer

On June 15, 2020, the U.S. FDA approved Jazz Pharmaceuticals and PharmaMar’s Zepzelca™ (lurbinectedin) for adult patients who have metastatic small cell lung cancer (SCLC) with disease progression during or after platinum-based chemo. SCLC accounts for approximately 15% of all lung cancers, or roughly 30,000 new patients in the U.S., annually. While it is generally more responsive to chemo and radiation than other types of lung cancer, the disease often is widely disseminated by the time of diagnosis -- making a cure difficult. Zepzelca, an alkylating agent, is administered as an IV infusion over 60 minutes. The recommended dose is 3.2 mg/m2 once every 21 days until disease progression or unacceptable toxicity. In a phase 2, single-arm, open-label trial, 105 patients received Zepzelca as a second-line treatment of SCLC. The primary endpoint, the overall response rate (ORR), was 35% with an average duration of response of 5.3 months. Zepzelca was approved through the FDA’s Accelerated Approval process, which requires additional clinical trial results confirming the preliminary data that show effectiveness. It was given Priority Review and Orphan Drug status, as well.

Jazz will market Zepzelca beginning in early July. Pricing is not known at this time. For full prescribing information, look here.

FDA Approves Lyumjev

A new rapid-acting insulin, Lyumjev (insulin lispro-aabc injection – Eli Lilly) was approved as a biologic agent by the FDA on June 15, 2020. Given SC immediately before or within 20 minutes of beginning meals, it is indicated to improve blood sugar management for adults who have type 1 or type 2 diabetes. Lyumjev containing a concentration of 100 Units/mL will be available in 10mL, multi-use vials and 3mL cartridges for the HumaPen® Luxura® HD insulin delivery device. Different types of prefilled, single-user, 3mL pens that contain either 100 Units/mL or 200 Units/mL also will be marketed. No launch date has been announced. The wholesale acquisition cost (WAC) will be the same as Humalog’s, but both products are included in the Lilly Insulin Value Program, which caps the monthly out-of-pocket cost of insulin at $35 for patients who have commercial insurance and those who have no insurance. Look here for its prescribing information.

New Indication for Cosentyx

Cosentyx® (secukinumab) injection, an interleukin-17 (IL-17) inhibitor manufactured by Novartis, was FDA approved on June 16, 2020, for treating non-radiographic axial spondyloarthritis (nr-axSpA). An inflammatory arthritis of the spine that usually is diagnosed in the late teens to mid-forties for patients who have chronic, severe back pain, nr-axSpA is estimated to affect over one million Americans. A progressive condition, it is hard to diagnose because x-rays do not show it. To treat nr-axSpA, a 150mg dose of Cosentyx will be injected SC once a week for four weeks and then once every four weeks. Some patients may not need the weekly lead-in dosing. Already approved to treat psoriasis, psoriatic arthritis and ankylosing spondylitis, Cosentyx is available as single-use pen devices and single-use prefilled syringes, as well as in vials of powder that must be reconstituted for use. Another IL-17 inhibitor, Taltz® (ixekizumab – Eli Lilly and Company) injection, was granted an indication for nr-axSpA on May 29, 2020.  Prescribing information for Cosentyx is here.

Ilaris Receives New Indication

Another Novartis product, Ilaris® (canakinumab), also was granted an additional indication on June 16, 2020. It now is FDA approved for the treatment of Still’s disease. Believed to be a variant of systemic juvenile idiopathic arthritis (SJIA), Still’s disease is an inflammatory arthritis associated with fever, joint pain and rash. Since its first FDA approval for cryopyrin-associated periodic syndrome (CAPS) in 2009, Ilaris has received additional indications for other types of periodic fevers and for SJIA. A monoclonal antibody that inhibits interleukin-1 beta (IL-1β), it is injected SC to decrease inflammation. Recommended dosing for Still’s disease is 4mg/kg by SC injection once every four weeks for patients who are at least two years old and who weigh at least 7.5kg (16.5 pounds). The maximum dose for any one injection is 300mg. Ilaris is dispensed in single-use vials containing 180mg of active drug after being mixed with 1mL of sterile preservative-free water. A Medication Guide supplied with Ilaris warns that patients using it have a higher than normal risk of developing serious infections. Here is prescribing information.

Expanded Pediatric Indication for Mylotarg

Wyeth Pharmaceuticals, now a subsidiary of Pfizer, was given FDA approval on June 16, 2020, for Mylotarg (gemtuzumab ozogamicin) to treat children as young as one month old for newly-diagnosed, CD33-positive acute myeloid leukemia (AML). Previously, it was limited to patients aged two years and older. A rare and aggressive cancer of the blood and bone marrow, AML is diagnosed for approximately 20,000 patients each year in the United States. Although adults account for the large majority of cases, up to 4% are among children and teens. Mylotarg combines gemtuzumab, an antibody specific for CD33, and ozogamicin, which attacks and destroys AML. Administered by a two-hour IV infusion, the recommended dose of Mylotarg for babies and children is either 0.1mg/m2 or 3mg/m2, depending on body surface area (BSA). Dosing is scheduled by the phase of treatment and by whether other cancer drugs are being used. A boxed warning cautions that using Mylotarg may cause potentially fatal damage to the liver. For its complete prescribing information, check here.

New Keytruda Indications

Keytruda® (pembrolizumab – Merck), a PD-1-blocking antibody that is administered as 30-minute IV infusions, was granted three new indications during June. First, it was given a second non-organ specific indication on June 16, 2020. As monotherapy, it now can be used to treat patients, including children, who have metastatic and/or inoperable solid tumors with tumor mutational burden-high (TMB-H) [≥10 mutations/megabase (mut/Mb)]. Patients will be tested for the mutation; previous treatment must no longer be adequate; and no other therapy options should be available before Keytruda is used. For this new indication, the adult dose is 200mg once every three weeks or 400mg once every six weeks. Pediatric patients will be given 2mg/kg with a maximum of 200mg/dose once every two weeks. On June 24, 2020, the FDA approved Keytruda to treat adults who have cutaneous squamous cell carcinoma (cSCC), who are not candidates for surgical or radiation therapy and whose cancer has metastasized or has returned after prior treatment. For cSCC, the recommended doses are 200mg once every three weeks or 400mg once every six weeks for up to 24 months or until the drug stops working or the patient cannot stand the side effects. Keytruda’sthird new indication was received from the FDA on June 29, 2020. It now can be used by itself as initial therapy for adults who have microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer that cannot be removed surgically or that has recurred. After the tumors have tested positive for the targeted mutations, dosing will be at either 200mg once every three weeks or 400mg once every six weeks. Keytruda has multiple prior indications that include numerous organ-specific cancers -- such as some esophageal, head and neck, kidney, lung and stomach cancers. It also is approved for some patients who have Merkel cell carcinoma (MCL), multiple myeloma and non-Hodgkin lymphoma (NHL) and some cancers that are grouped by genetic mutation, not by the affected organ. For Keytruda’s updated prescribing information look here.

Tazverik Gains Additional Indications

On June 18, 2020, the FDA approved Tazverik (tazemetostat – Epizyme) tablets, under Priority Review, for the treatment of adults who have one of two separate types of follicular lymphoma (FL). The first new indication is for treatment of patients who have relapsed or refractory FL, whose tumors are positive for a specific enzyme mutation -- enhancer of zeste homolog 2 (EZH2) -- and who have received at least two prior systemic therapies. The second is for patients who have relapsed or refractory FL that has no alternative treatment options. Tazverik originally was approved on Jan. 23, 2020, for treatment of patients 16 years and older who have metastatic or locally advanced epithelioid sarcoma and who are not eligible for surgery. Accelerated Approval for the new indications was based on data from Phase 2 studies that included nearly 100 patients who have FL that worsened despite two or more prior treatments. With an ORR of 69%, the patients who have an EZH2 activating mutation averaged a duration of response (DOR) of 10.9 months when treated with Tazverik. For the study participants who have wild-type EZH2, the ORR was 34% and the average DOR was 13 months. The recommended dose of Tazverik is 800mg orally twice daily with or without food until disease progression or unacceptable toxicity. Hereis full prescribing information.

FDA Approves Crysvitafor Tumor-Induced Osteomalacia

The FDA approved Ultragenyx and Kiowa Kirin’s Crysvita® (burosumab-twza) on June 18, 2020, to treat fibroblast growth factor 23 (FGF23)-related hypophosphatemia (low blood levels of phosphate) due to tumor-induced osteomalacia (TIO) that is associated with inoperable or localized phosphaturic mesenchymal tumors. The new approval includes adults and pediatric patients two years of age or older. TIO causes softening of the bones, bone lesions, low levels of phosphate in the blood, bone pain, fractures and weakness. It occurs because overly high levels of FGF23 cause the kidneys to excrete excess phosphate and reduce vitamin D production. Crysvita works by blocking FGF23 -- allowing for increased production of vitamin D and increased intestinal absorption of phosphate and calcium, which are important for bone strength. Around 500 to 1,000 people in the U.S. suffer from TIO. About one-half have inoperable tumors, with the only treatment consisting of vitamin D and phosphate supplementation. Approved under Priority Review, this second follows its original approval on April 18, 2018, for the treatment of X-linked hypophosphatemia (XLH) in adults and pediatric patients at least one year of age. Later the approval was extended to patients at least six months old. The new indication, which received Priority Review, is based on phase 2 study results showing that patients achieved increased serum phosphorus and vitamin D levels. Bone scans also indicated bone lesions were healing for treated patients. Before beginning Crysvita, patients should discontinue any oral phosphate or active vitamin D analog products. A healthcare provider administers Crysvita SC and monitors serum phosphorus levels. The pediatric starting dose is 0.4mg/kg, which can be adjusted by rounding to the nearest 10mg, given once every two weeks. Adult doses, which start at 0.5mg/kg once every four weeks, may increase by 2mg/kg and shift to once every two weeks, if needed. Single doses should not exceed 180mg every two weeks for either age group. For full prescribing information see here.

New Delivery Device for Dupixent

The FDA approved a 300mg prefilled pen device for Dupixent® (dupilumab) injection on June 18, 2020. Jointly developed by Sanofi and Regeneron, Dupixent is an interleukin-4 alpha receptor (IL-4Rα) blocking antibody that disrupts the inflammatory process. Injected SC, it is indicated to treat atopic dermatitis for patients six years old and older, with dosing determined by the patient’s weight. For patients as young as 12 years old, it also is approved for treating eosinophilic or oral steroid dependent asthma at a dose of 200mg or 300mg once every two weeks after one larger loading dose. Dupixent’s third FDA indication is at 300mg once every two weeks to treat patients aged 18 and older for chronic rhinosinusitis with nasal polyposis. Intended for patients at least 12 years old, the new single-dose auto-injector has both written and spoken directions for use. Its launch is planned for third quarter 2020. Previously available prefilled 200mg and 300mg syringes will continue to be marketed and a 200mg pen is under FDA review. Look here for full prescribing information

Gimoti FDA Approved

A new formulation, Gimoti (metoclopramide – Evoke Pharma) nasal spray, was approved by the FDA on June 19, 2020. It is indicated to treat adults who have gastroparesis resulting from diabetes. Gastroparesis, which can be acute or chronic, is slowed emptying of stomach contents, often associated with diabetes and other conditions, such as anorexia, infections and stomach surgeries. Symptoms include abdominal bloating and pain, constipation or diarrhea, heartburn, nausea and vomiting. With women accounting for approximately 80% of patients, diabetes-related gastroparesis is believed to affect as many as 16 million U.S. adults. As the only drug FDA approved to treat gastroparesis, metoclopramide is available in oral solid and liquid forms, as well as injectables. Oral forms may not be absorbed adequately for full relief, though; and the injectables are administered either IV or IM, making them difficult for patients to use. Metoclopramide works by promoting muscle contractions but not secretions in the stomach. Gimoti is the first nasal form, which will be dispensed in 9.8mL bottles that contain about 120 sprays each containing 15mg of active drug. Directions are to use one spray one-half hour or more before each meal and at bedtime. According to a boxed warning on their labeling, metoclopramide products should not be used for longer than 12 weeks because they may cause tardive dyskinesia (TD). A serious and potentially permanent condition, TD may produce uncontrollable arm and leg jerking, blinking, lip smacking and other muscle movements. Through its partner, Eversana, Evoke plans a launch in the fourth quarter of 2020. Here is prescribing information for Gimoti.

New Xpovio Indication

Karyopharm Therapeutics received a second FDA indication on June 22, 2020, for its selective inhibitor of nuclear export (SINE), Xpovio® (selinexor) tablets. The new approval is to treat not-otherwise-specified diffuse large B-cell lymphoma (DLBCL) for adults who have been treated at least twice, but whose cancer recurs or resists previous therapy. To treat DLBCL, Xpovio will be taken on the first and third days of each week. In the phase II clinical study leading to the new indication, 18 of the 134 participants achieved a complete response and 21 others responded partly. Responses lasted six months for 38% of the responding patients. The only currently approved drug in its class, Xpovio works by preventing the transfer of specific growth-promoting and tumor-repressing proteins from cell nuclei. Its initial U.S. approval was two years ago, in combination with dexamethasone, to treat multiple myeloma. Because the DLBCL indication was awarded under the FDA’s Accelerated Approval program, confirmation of safety and effectiveness will need to be proven in further studies before full approval is given. Check here for its complete prescribing information.

FDA Approves Fintepla for Dravet Syndrome

Fintepla® (fenfluramine) oral solution was approved by the FDA on June 26, 2020. A C-IV controlled substance, it will be used for treating patients who have Dravet syndrome (DS), a rare form of epilepsy that typically is diagnosed in infancy. To treat DS, patients at least two years old will be given 0.1mg/kg two times a day. The dose can be increased weekly up to 0.35mg/kg twice daily with a daily limit of 26mg, even if the patient is taking other medications for DS. However, Fintepla doses are reduced when stiripentol plus clobazam are being used; the maximum daily maintenance dosage of Fintepla for patients taking these medications is 0.2 mg/kg twice daily (maximum daily dosage of 17 mg). It will be available as a color-free, cherry-flavored solution which should be discarded three months after opening. A boxed warning outlines its potential to cause valvular heart disease and/or pulmonary arterial hypertension (PAH). Additionally, a risk evaluation and mitigation strategy (REMS) is in place to certify prescribers, dispensers and patients. Zogenix expects to introduce Fintepla in July at a wholesale acquisition cost (WAC) of $96,000 per year of therapy. Two other drugs, Diacomit® (stiripentol - Biocodex) and Epidiolex® (cannabidiol - GW Pharmaceuticals) were FDA approved in 2018 for DS. Diacomit must be used in combination with Onfi® (clobazam - Lundbeck) and using more than one drug at a time is not unusual for treating DS. Prescribing information for Fintepla is here.

Mycapssa - First Oral Acromegaly Drug Approved

Mycapssa® (octreotide) capsules was approved by the FDA on June 26, 2020. The first oral drug of its type, it is indicated as maintenance for patients who have acromegaly that has been treated successfully with another octreotide product or with Somatuline®Depot (lanreotide - Ipsen). Acromegaly, most frequently caused by nonmalignant pituitary tumors, produces characteristic broadening of the face, feet and hands. It also may be associated with bone, heart and lung conditions. Estimated to affect between 40 and 130 individuals per million, acromegaly typically is diagnosed for adults in middle age -- about equal numbers of men and women. Children also may be affected, however; and diagnosis for any age group may be delayed due to slow onset of symptoms. Usually resulting from non-cancerous growths on the pituitary gland, acromegaly elevates the amounts of growth hormone (GH) in the body. Levels of another protein, insulin-like growth factor 1 (IGF-1), can also be higher than normal ranges. Octreotide is an analogue of somatostatin, a naturally produced hormone that regulates the production of GH, insulin and other secretions. If needed, Mycapssa’s recommended initial dose of 20mg twice a day may be increased to a daily maximum of 80mg (two capsules twice a day). The manufacturer, Chiasma, plans a fourth quarter 2020 launch at a wholesale acquisition cost (WAC) of $5,152 for a 28-day supply of capsules at the 40mg/day dose level. For complete prescribing information, check here.

Genentech Receives FDA Approval for Phesgo

Phesgo (pertuzumab/trastuzumab/hyaluronidase-zzxf) injection was approved by the FDA on June 29, 2020. It is indicated, along with other drugs, to treat human epidermal growth factor receptor 2 positive (HER2+) breast cancer. A fixed-dose combination of two monoclonal antibodies that each obstruct separate parts of HER2 receptors, it is the first SC formulation for either of its active components. Additionally, it contains a proprietary hyaluronidase that is formulated to allow rapid SC administration. Although Phesgo still must be administered by a healthcare professional, treatments will need only a few minutes compared to one hour or longer for the IV infused dosage forms of the two drugs. Patients also can be treated at home instead of in a healthcare facility. Genentech’s IV formulation of pertuzumab is available separately as the brand Perjeta®. Its IV trastuzumab is marketed as the brand Herceptin®, which has four biosimilars. None of the previously available products can be interchanged with Phesgo, which will be available in single-dose vials that contain either pertuzumab 600mg/trastuzumab 600mg/hyaluronidase 20,000 Units or pertuzumab 1,200mg/trastuzumab 600mg/hyaluronidase 30,000 Units. One loading dose will be given at the higher strength of pertuzumab, then all subsequent doses will use the lower strength combination once every three weeks. Boxed warnings caution that using Phesgo may result in serious adverse effects on the heart and/or lungs. Because it also can damage an unborn baby, women of childbearing age who are receiving it should use reliable contraception during and for at least seven months following treatment. Launch and pricing plans have not been announced. Phesgo will be added to Express Scripts’ specialty drug list. Go here for full prescribing information.

New Bavencio Indication

The FDA has approved Bavencio® (avelumab – EMD Serono/Pfizer) injection to maintain disease-free status for patients whose locally advanced or metastatic urothelial carcinoma has not progressed during chemo with a platinum compound. The new maintenance indication, which was granted on June 30, 2020, is based on results of a phase III clinical trial showing that OS for patients using Bavencio along with best supportive care averaged about seven months longer than for patients receiving only best supportive care (21.4 months as opposed to 14.3 months). Bavencio is a PD-)-blocking antibody that augments the immune system’s ability to destroy cancer cells. Its recommended dosing is 800mg given as a 60-minute IV infusion once every two weeks. It has additional approvals as monotherapy to treat metastatic Merkel cell carcinoma (MCC) and with Inlyta® (axitinib - Pfizer) tablets for advanced renal cell carcinoma (RCC). The new indication for Bavencio was expedited under the FDA’s Breakthrough Therapy and Real-Time Oncology (ROTR) programs. Its previous Accelerated Approval to treat patients whose urothelial carcinoma is worsening after chemo has now been upgraded to a full FDA approval, as well. Check here for its complete revised prescribing information. Dojolvi Approved for Long-Chain Fatty Acid Oxidation Disorders

FDA Approves Dojolvi

On June 30, 2020, Ultragenyx received approval from the FDA for Dojolvi (triheptanoin) oral liquid as a source of calories and fatty acids for the treatment of pediatric and adult patients who have molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). A group of genetic disorders, LC-FOAD prevents the body from converting long-chain fatty acids into energy. Approximately 2,000 to 3,500 adults and children in the United States have LC-FAOD. Due to the body’s inability to produce energy from fat, patients with LC-FAOD can experience severe complications including early death. Current treatment options include avoidance of fasting, a low-fat/high carbohydrate diet, carnitine and medium-chain triglyceride (MCT) oil, which is a medical food. Dosing for Dojolvi is based on the patient’s metabolic requirements as determined by daily caloric intakes (DCI). The recommended target daily dose is up to 35% of a patient’s total prescribed DCI divided into at least four doses; diluted with foods, liquids or formula; and administered through a feeding tube. Ultragenyx plans on launching Dojolvi within 30 days. For full prescribing information, look here.

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