FDA Update: September 2018

Sep 18, 2018
New Pill Bottle

First Generic for Adcirca Approved

Mylan Pharmaceuticals received FDA approval on Aug. 3, 2018, for tadalafil tablets, 20mg, the first generic for Adcirca® (United Therapeutics). A phosphodiesterase type 5 (PDE5) inhibitor, tadalafil is taken once a day to treat pulmonary arterial hypertension (PAH). Affecting about 50,000 Americans, PAH is a rare, serious vascular disease that results from dangerously high pressure in the blood vessels leading from the heart to the lungs. Launch is planned before the end of August and Mylan will have six months of exclusivity before other generics can be marketed in the U.S. Annual U.S. sales for Adcirca are approximately $483 million.

Orkambi Granted Expanded Indication and New Dose Form

On Aug. 6, 2018, Orkambi® (lumacaftor/ivacaftor), which previously was approved to treat children age six and older, received FDA approval for children as young as two years of age. Additionally, it will be available as oral granules -- making dosing easier for children who may not be able to swallow a tablet. The combination works in two ways to correct cystic fibrosis-causing mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Lumacaftor is a CFTR corrector, which helps CFTR proteins reach the cell surface. Ivacaftor is a CFTR potentiator, which helps keep the CFTR protein channels on the cell surface open longer to increase the flow of salt and water into and out of the cell. Granule packets will be marketed in two strengths, lumacaftor 100mg/ivacaftor 125mg (for children who are between two years and six years old and who weigh less than 14 kg or about 30 pounds) and lumacaftor 150mg/ivacaftor 188mg (for children in the same age group who weigh more than 14 kg). One packet of granules will be mixed with one teaspoonful of a liquid or a soft food and given along with food that contains a high amount of fat. Doses are given 12 hours apart. Vertex, which plans to launch the granule packets within the next month, estimates that about 1,300 more children may now be eligible for Orkambi treatment. Complete, revised prescribing information is available here.

Poteligeo Approved for Rare Non-Hodgkin Lymphomas

Poteligeo® (mogamulizumab-kpkc) injection for IV use was approved by the FDA on Aug. 8, 2018. It is a monoclonal antibody that attaches to CC chemokine receptor type 4 (CCR4), a protein on the surfaces of some cancer cells. Its indications are for adults who have had one or more systemic treatments for mycosis fungoides (MF) or Sézary syndrome (SS), but whose cancers have returned or have resisted therapy. MF and SS are rare variants of non-Hodgkin lymphoma that affect the skin. Poteligeo is dosed by weight at 1mg/kg and given as IV infusions on 28-day cycles. For the first cycle, it will be administered four times at one-week intervals; then treatments are reduced to one infusion every two weeks for the following cycles. Dosing continues until the cancer worsens or the patient cannot tolerate the drug’s side effects. Kyowa Kirin is planning a fourth quarter 2018 launch. Complete prescribing information currently is available here.

FDA Approves Jornay PM to Treat ADHD

On Aug. 8, 2018, the FDA approved Jornay PM (methylphenidate) extended-release capsules to treat attention deficit hyperactivity disorder (ADHD). Indicated for use by children age six and older, it utilizes Ironshore Pharmaceuticals’ patented Delexis® technology, which slows down drug absorption. Like all methylphenidates, it is a C-II controlled substance. Unlike other methylphenidate products, however, Jornay PM is not taken in the morning. The recommended initial dose is 20mg taken once a day at about 8:00 p.m. Gradual overnight release of the drug manages morning ADHD symptoms and then lasts throughout the day. Doses may be adjusted on a weekly basis to a maximum of 100mg per day. Timing also may be changed to between around 6:30 and 9:30 in the evening, depending on the child’s response. A boxed warning on the labeling cautions that all central nervous system (CNS) stimulants, including methylphenidate, carry risks for abuse and dependence. Complete prescribing information is here.

Onpattro Approved for Hereditary Transthyretin-Mediated Amyloidosis

The FDA approved Alnylam’s Onpattro (patisiran) lipid complex injection on Aug. 10, 2018, for the treatment of adults with peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR). Onpattro works by blocking (silencing) mutated ribonucleic acid (RNA) that causes hATTR, a rare disease that produces disability, pain and organ damage. It is the first FDA-approved ribonucleic acid interference (RNAi) drug and the first pharmacological treatment specifically for hATTR. Given by IV infusion once every three weeks, Onpattro is dosed at 0.3mg/kg for patients weighing less than 100kg (about 220 pounds). Patients weighing more will receive a fixed dose of 30mg. Alnylam plans an immediate launch. Complete prescribing information is available here.

Annovera, Vaginal Birth Control Approved

The Population Council, Inc. received FDA approval for Annovera (segesterone acetate/ethinyl estradiol vaginal system) on Aug. 10, 2018. It combines a new progestin (segesterone acetate) with a commonly used estrogen to provide hormonal contraception for one year. A ring-shaped vaginal insert, it is placed in the vagina by the user. It remains in place, releasing low levels of both hormones, for 21 days, then it is removed, washed and stored in a special case for one week. It can be used for up to 13 cycles (one year). The Population Council is a nonprofit, international organization that promotes public health solutions in developing countries. Through a partnership with TherapeuticsMD, Annovera will be available in the United States. Its full prescribing information is here.

Galafold Approved to Treat Fabry Disease

Galafold (migalastat – Amicus Therapeutics) was approved by the FDA on Aug. 10, 2018. It is indicated to treat adults who have Fabry disease that has been identified by a diagnostic test as resulting from one or more of multiple abnormalities in the galactosidase alpha (GLA) gene. The first oral treatment for Fabry disease, it also is the first FDA-approved alpha-galactosidase A (α-Gal A) chaperone (enhancer). It boosts the activity of the patient’s existing α-Gal A, an enzyme that is lacking and/or dysfunctional in Fabry disease. Previously available treatment replaces missing α-Gal A. Recommended dosing is one oral capsule every other day at least two hours before and two hours after any food. Galafold was launched upon approval. Complete prescribing information for Galafold is available here.

Cequa Approved to Treat Dry-Eye Disease

Sun Pharmaceuticals’ Cequa (cyclosporine ophthalmic solution) 0.09% received approval from the FDA on Aug. 14, 2018. An immunosuppressant in the calcineurin inhibitor class, it is indicated for topical use to increase the production of tears for patients who have keratoconjunctivitis sicca (dry eyes). It is a preservative-free formulation that uses unique nanomicelles to improve solubility and increase diffusion into the eye. Cequa also is a higher strength than other cyclosporine eye drops. Recommended dosing is one drop in each eye twice a day at 12-hour intervals. It will be dispensed in boxes of 60 single-use vials containing 0.25mL (one dose for each eye). Sun Pharmaceuticals has not disclosed its launch or pricing plans. Prescribing information is here.

Kalydeco’s Indication Expanded

Vertex Pharmaceuticals’ Kalydeco® (ivacaftor) was approved by the FDA on Aug. 15, 2018, to treat children as young as one year old and who weigh at least 7kg (approximately 15 pounds). Previously, its use was limited to children age two years and older. Kalydeco treats cystic fibrosis (CF) caused by mutations in the CFTR gene. Before treatment begins, patients must test positive for susceptible mutations. For children who weigh less than 14kg (around 30 pounds), recommended treatment is one 50mg dose every 12 hours with a food that contains fat. The dose increases to 75mg twice a day for children weighing more than 14kg. Kalydeco is available in 50mg and 75mg single-dose pouches of granules to mix with about a teaspoonful of liquid or soft food for administration. It is also supplied as 150mg tablets for adults and older children. Its complete prescribing information is available here.

First True Generic to EpiPen Approved

Teva Pharmaceutical Industries was granted FDA approval on Aug. 16, 2018, for its AB-rated generic versions of Mylan’s EpiPen® (epinephrine) Auto-Injector 0.3mg and EpiPen, Jr® (epinephrine) Auto-Injector 0.15mg. Used as emergency treatment for anaphylaxis (severe, systemic allergic reactions), they are indicated for adults and children who weigh at least 33 pounds. Recommended use is one auto-injection into the middle of the outer thigh (through clothing, if necessary) at the onset of the reaction. A second injection may be needed before emergency personnel arrive, but no more than two doses should be self-injected for one episode. Teva is expected to launch its generic epinephrine auto-injector in the coming months. Pricing information is not yet available. U.S. sales for EpiPen and EpiPen, Jr totaled approximately $763 million in 2017.

Diacomit Approved to Treat Dravet Syndrome

Biocodex received FDA approval for Diacomit (stiripentol) on Aug. 20, 2018. It is indicated to treat seizures for patients who have Dravet Syndrome (DS), who take Onfi® (clobazam - Lundbeck) and who are at least two years old. Recommended dosing is 50mg/kg daily, divided into two or three doses and taken along with a meal. Capsules should be swallowed whole; powder packets should be mixed into 100mL (about three ounces) of water to drink. Because Diacomit may cause neutropenia and/or thrombocytopenia, the patient’s blood counts should be checked before they begin treatment and then every six months during therapy. It will be dispensed with a Medication Guide. Launch date, pricing and distribution plans are not known at this time. Complete prescribing information is here.

Oxervate approved for Neurotrophic Keratitis

On Aug. 22, 2019, the FDA approved Oxervate (cenergermin-bkbj ophthalmic solution) a breakthrough-designated treatment for neurotrophic keratitis. A rare, degenerative disease of the cornea, it affects fewer than 50 individuals in 100,000. Resulting from the loss of corneal sensitivity, it can progressively damage the top layer of the cornea. Ulcerations and perforations can occur in severe cases. The recommended dose is one drop into the affected eye(s) six times a day, at two hour intervals, for a total of eight weeks. Oxervate, a product of Dompé farmaceutici SpA, is the first human nerve growth factor to be approved as a drug and it also is the first topical biologic eye therapy. Oxervate will be made available in the United States by early 2019. Pricing is not available at this time. Complete prescribing information is available here.

Inveltys, New Ophthalmic Corticosteroid, Approved

Also on Aug. 22, Kala Pharmaceuticals received FDA approval for Inveltys (loteprednol etabonate ophthalmic suspension) 1%. It is indicated to treat pain and inflammation after eye surgery. Starting one day after the surgery and for the following two weeks, one or two drops should be put into the affected eye(s) two times a day. A proprietary technology, mucus-penetrating particles, helps Inveltys infiltrate better into the eyes than other corticosteroid eye drops, which usually are needed four times a day. Because extended use of an ophthalmic corticosteroid can cause glaucoma or damage optic nerves, intraocular pressure (IOP) should be checked if the drug is used for 10 days or longer. Kala plans an early 2019 launch. Prescribing information is here.

Takhzyro Approved to Prevent Hereditary Angioedema Attacks

Shire was given FDA approval for Takhzyro (lanadelumab -flyo) on Aug. 23, 2018. Takhzyro is indicated to treat hereditary angioedema (HAE) for patients at least 12 years old. The first monoclonal antibody to be approved for HAE therapy, it blocks plasma kallikrein to prevent swelling caused by HAE. Recommended initial dosing is one 300mg subcutaneous (SC) self-injection every two weeks, which may be reduced to once every four weeks if the patient has no attacks for six months. Complete prescribing information is here.

Xerava Approved to Treat Abdominal Infections

The FDA approved Xerava (eravacycline) on Aug. 27, 2018, to treat complicated (extensive) intra-abdominal infections in patients at least 18 years of age. It is a tetracycline antibiotic administered as an intravenous (IV) infusion. Recommended dosing is one mg/kg of body weight every 12 hours for four days to 14 days. It can be used to treat multiple infection types, including appendicitis, cholecystitis and peritonitis, that are caused by bacteria known or believed to be sensitive to Xerava’s effects. The manufacturer, Tetraphase Pharmaceuticals, plans a launch in the fourth quarter of 2018. Prescribing information is here.

Hemophilia A Drug, Jivi, Approved

Bayer’s Jivi® (antihemophilic factor [recombinant] PEGylated-aucl) was FDA approved for treating hemophilia A, on Aug. 30, 2018. It is a coagulation factor VIII replacement indicated for use to prevent bleeds in patients who are at least 12 years old and who have had previous treatment. Jivi also has FDA indications to treat active bleeding and to manage bleeding associated with dental and surgical procedures. For bleeding prevention, the recommended dosing is based on the patient’s weight at 30IU to 40IU/kg two times per week. Doses and dose intervals may be adjusted according to the patient’s response, and acute doses may be added to control bleeds. Bayer plans to begin shipping Jivi in the first week of September. Full prescribing information for Jivi is here

FDA Approves Delstrigo and Pifeltro for HIV

Merck received FDA approval for Delstrigo (doravirine/lamivudine/tenofovir disoproxil fumarate) tablets and Pifeltro (doravirine) tablets on Aug. 30, 2018. Both are once-daily treatments for adults who have HIV, but who have not had prior treatment with an antiretroviral medication. Delstrigo, which combines Pifeltro, a new non-nucleoside reverse transcriptase inhibitor (NNRTI), with two nucleoside reverse transcriptase inhibitors (NRTIs), will be taken alone. Its labeling includes a boxed warning that discontinuing the use of lamivudine or tenofovir disoproxil fumarate can activate hepatitis B virus (HBV) for patients who have or who have had HBV. Patients should be monitored for signs of HBV before, during and for several months after treatment with Delstrigo. Pifeltro must be used along with other antiretrovirals. Merck expects that both drugs will be available within the month. Full prescribing information for Delstrigo is here. Pifeltro prescribing information is here.  

Imbruvica Plus Rituxan Approved for Waldenström’s Macroglobulinemia

On Aug. 24, 2018, the FDA approved Imbruvica® (ibrutinib – Pharmacyclics/Janssen) together with Genentech’s Rituxan® (rituximab) to treat Waldenström’s macroglobulinemia (WM), a rare type of non-Hodgkin lymphoma. Imbruvica is a Bruton’s kinase inhibitor (BKI) that works by blocking a specific enzyme needed by the cancer to multiply and spread. For WM, its recommended dose is 420mg orally once a day. Rituxan, a CD20-directed cytolytic antibody approved to treat several different cancers, is used off-label for several other cancers, including WM. It is given as an IV infusion at 375mg/m2 once each week for four weeks followed by a three-month break and then infused for four more weekly doses. When both drugs are used on the same day, Imbruvica should be taken first. In the iNNOVATE Study, progression-free survival (PFS) lasted at least 30 months for 82% of dual therapy patients, but for only 28% of those treated with Rituxan and a placebo. Imbruvica’s complete prescribing information is here

Sodium-Glucose Cotransporter-2 Inhibitors

The FDA issued a warning on Aug. 29, 2018, about a rare but potentially severe side effect from taking sodium-glucose cotransporter-2 (SGLT2) inhibitors. The drugs, used to treat type 2 diabetes, can cause necrotizing fasciitis of the perineum, a potentially fatal infection of the genitals. Also called Fournier’s gangrene, the condition is known to have hospitalized at least 12 patients taking a SGLT2 inhibitor in the last five years. Patients who take an SGLT2 drug and experience a fever along with redness, swelling and/or pain in the genital area should get immediate, urgent medical care. All FDA-approved SGLT2 inhibitors will carry additional warnings about the condition on their labeling and in their Medication Guides for patients. For more information, including a list of SGLT2 inhibitors approved in the U.S., please see the FDA notice here.




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