2014 FDA drug news in endocrinology

In 2014, Endocrine Today provided the latest information on drugs related to the field that were newly approved, cleared and had label or indication changes.

Below we present a timeline of the most important FDA approvals in 2014 covered by Endocrine Today. 

FDA approves dapagliflozin for type 2 diabetes

The FDA approved dapagliflozin (Farxiga, Bristol-Meyers Squibb and AstraZeneca), according to a press release. This is the second sodium-glucose co-transporter 2 inhibitor approved for the treatment of type 2 diabetes.

“Controlling blood sugar levels is very important in the overall treatment and care of diabetes, and Farxiga provides an additional treatment option for millions of Americans with type 2 diabetes,” Curtis Rosebraugh, MD, MPH, director of the Office of Drug Evaluation II in the FDA’s Center for Drug Evaluation and Research, said in a press release. Read more.

FDA approves Myalept for treatment of lipodystrophy

The FDA approved metreleptin, in combination with diet, for the treatment of complications of leptin deficiency in patients with congenital generalized or acquired generalized lipodystrophy, according to a press release.

“Myalept is the first approved therapy indicated for treating the complications associated with congenital or acquired generalized lipodystrophy and provides a needed treatment option for patients with this orphan disease,” Mary Parks, MD, deputy director of the Office of Drug Evaluation II in the FDA’s Center for Drug Evaluation and Research, said in the release. Read more.

FDA approves Vimizim for Morquio A syndrome

The FDA approved elosulfase alfa, an enzyme replacement treatment for patients with mucopolysaccharidosis type IVA, or Morquio A syndrome. According to a press release from the manufacturer, it is being approved with a boxed warning to include the risk for anaphylaxis.

Elosulfase alfa (Vimizim, BioMarin Pharmaceutical) is the first FDA-approved enzyme replacement therapy to target the underlying causes of the rare disease, which affects approximately 3,000 patients in the developed world. It also is the first agent to receive the rare pediatric disease priority review voucher. Morquio A syndrome, a type of lysosomal storage disorder, affects bone development, growth and mobility. Read more.

FDA approves injectable testosterone for hypogonadism

Endo Pharmaceuticals announced that the FDA has approved its testosterone undecanoate for hypogonadism, according to a company press release.

The approval comes after an 84-week phase 3 trial in the US in men diagnosed with hypogonaism, with an average age of 54 years and total serum testosterone levels less than 300 ng/dL. Data from the study demonstrated testosterone undecanoate (Aveed, Endo Pharmaceuticals) increased and maintained mean serum testosterone levels for up to 10 weeks. Read more.

FDA expands thyroid drug use

The FDA announced revised prescribing information for thyrotropin alfa, an injectable drug given prior to radioactive iodine treatment for thyroid cancer.

Injectable thyrotropin alfa (Thyrogen, Genzyme) was recommended for prescription for patients receiving 100 mCi of radioactive iodine (RAI) treatment, but has been expanded to include patients given doses between 30 mCi and 100 mCi. The revised prescribing information follows two studies published in the New England Journal of Medicine in May 2012 comparing ablation outcomes among patients who received recombinant human thyrotropin with patients who underwent thyroid hormone withdrawal at both low and high doses of RAI, according to a company press release. Those who received thyrotropic alfa were less likely to have hypothyroid symptoms. Read more.

FDA approves Tanzeum to improve glycemic control in type 2 diabetes

The FDA announced that it has approved albiglutide subcutaneous injection to improve glycemic control in adult patients with type 2 diabetes.

Albiglutide (Tanzeum, GlaxoSmithKline), a glucagon-like peptide-1 receptor agonist, showed HbA1c improvement for more than 2,000 patients in a series of eight clinical trials. Read more.

FDA grants orphan drug status to Prader-Willi treatment

The FDA granted diazoxide choline, studied previously for high triglycerides, orphan drug status for the treatment of Prader-Willi syndrome. This drug is used in a diazoxide controlled release tablet known as DCCR.

“We greatly appreciate the FDA’s support of our efforts to evaluate the use of DCCR in the treatment of Prader-Willi syndrome. We are actively recruiting Prader-Willi syndrome patients for a recently initiated clinical study. Initial results from that study should be coming out during Q3 of this year,” Neil M. Cowen, PhD, president and chief scientific officer of Essentialis said in a press release from the company. Read more.

FDA approves nasal testosterone therapy

The first testosterone nasal gel for replacement therapy in adult men has been approved by the FDA for use in conditions associated with a deficiency or absence of endogenous testosterone, according to the manufacturer.

Natesto (testosterone, Trimel Pharmaceuticals Corporation) — an androgen intended to treat patients with primary or hypogonadotropic hypogonadism, congenital or acquired — is self-administered via a nasal applicator, minimizing the risks of secondary exposure to testosterone by women or children, according to the release. Read more.

FDA approves Afrezza inhaled insulin for adults with diabetes

The FDA approved Afrezza inhalation powder to improve glycemic control in adults with diabetes.

Afrezza (MannKind Corporation) is an inhaled insulin administered at the beginning of each meal. Read more.

FDA approves Eylea for treatment of diabetic macular edema

The FDA has approved Eylea for treatment of diabetic macular edema, according to a Regeneron Pharmaceuticals press release.

This is the third FDA-approved indication for the aflibercept injection, which was approved for treatment of neovascular age-related macular degeneration in 2011 and for treatment of macular edema following central retinal vein occlusion in 2012. Read more.

FDA approves canagliflozin/metformin combination tablet for type 2 diabetes

The FDA announced its recent approval of a canagliflozin/metformin combination tablet to reduce blood glucose in adults with type 2 diabetes.

Invokamet (canagliflozin/metformin HCl, Janssen Pharmaceuticals, Inc) pairs the sodium glucose co-transporter 2 (SGLT2) inhibiting action of canagliflozin (Invokana, Janssen) with metformin. Read more.

New insulin glargine awarded tentative FDA approval

The FDA has granted tentative approval for a new insulin glargine injection developed by Eli Lilly and Company and Boehringer Ingelheim to improve blood glucose control in patients with diabetes, according to a statement from the manufacturing alliance.

Basaglar, a basal insulin intended to provide long-lasting glycemic control between meals and during the night, is indicated to benefit adults with type 2 diabetes in conjunction with mealtime insulin and children with type 1 diabetes. Read more.

FDA approves Iluvien for treatment of DME

The FDA has approved Iluvien for the treatment of diabetic macular edema in patients previously treated with corticosteroids who did not have a significant increase in IOP, Alimera Sciences announced in a press release.

Iluvien (fluocinolone acetonide implant 0.19 mg) was approved with no requirement that patients undergo cataract surgery, according to the release. Read more.

FDA approves expanded indication for Ozurdex implant

The FDA has approved Ozurdex for the general patient population being treated for diabetic macular edema, Allergan announced in a press release.

In June, the FDA approved Ozurdex (dexamethasone intravitreal implant 0.7 mg) for the treatment of DME in pseudophakic adult patients or patients who were scheduled for cataract surgery. Read more.

FDA approves dulaglutide to treat type 2 diabetes

The FDA approved dulaglutide as a once-weekly injection for patients with type 2 diabetes to improve glycemic control in conjunction with diet and exercise.

Dulaglutide, a glucagon-like peptide-1 receptor to be known as Trulicity (Eli Lilly), has been studied as a solo treatment and in conjunction with other therapies such as metformin, sulfonylurea and others. In the most recent study published, dulaglutide was shown comparable to liraglutide (Victoza, Novo Nordisk) in controlling HbA1c and hypoglycemia. The most common side effects were nausea, diarrhea, vomiting, abdominal pain and decreased appetite. Read more.

FDA approves Contrave for chronic weight management

The FDA granted approval for naltrexone hydrochloride and bupropion hydrochloride extended-release tablets for adults to use, in conjunction with reducing calories and exercising, for chronic weight management, according to a statement from the agency. Naltrexone hydrochloride and bupropion hydrochloride (Contrave, Orexigen) is designed for adults with obesity (BMI ≥30) or adults who are overweight (BMI ≥27) with at least one weight-related condition including hypertension, type 2 diabetes or high cholesterol. Read more.

FDA approves Xigduo XR to treat adults with type 2 diabetes

The FDA approved a formulation combining two antihyperglycemic agents in a once-daily extended-release tablet to treat adults with type 2 diabetes, according to a statement from the drug developer.

The combination of dapagliflozin (Farxiga, Bristol-Myers Squibb/Astrazeneca) and metformin hydrochloride extended release (Xigduo XR, AstraZeneca) is designed for use with diet and exercise to improve glycemic control. Read more.

FDA approves Somatuline Depot for gastroenteropancreatic neuroendocrine tumors

The FDA approved lanreotide for treatment of patients with unresectable, well- or moderately differentiated, locally advanced or metastatic gastroenteropancreatic neuroendocrine tumors.

The approval was based in part on results of a trial that included 204 patients with non-functioning gastroenteropancreatic neuroendocrine tumors. More than half of the patients (55%) had tumors that were outside of the pancreas. Read more.

Signifor long-acting release wins FDA approval for acromegaly treatment

The FDA has approved a long-acting release formulation of pasireotide to treat patients with acromegaly who have either not responded to surgery or for whom surgery is not an option, according to statement from the drug developer.

Pasireotide long-acting release (LAR) (Signifor, Novartis), a next-generation somatostatin analog (SSA) designed for injectable suspension for intramuscular use, addresses an unmet need among patients with the endocrine disorder, according to the statement. Read more.

FDA approves Saxenda injection therapy for weight management

The FDA granted approval to liraglutide 3-mg injection as the first once-daily human glucagon-like peptide-1 analogue to help manage obese or overweight patients, according to a press release from the agency.

Liraglutide 3-mg injection (Saxenda rDNA origin, Novo Nordisk) is approved for adults with obesity with BMI ≥30 kg/m² or overweight adults with BMI ≥27 mg/m² and at least one weight-related comorbidity including hypertension, type 2 diabetes or high cholesterol dyslipidemia to use along with diet and physical activity, according to the press release. Read more.