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Monthly News Roundup - November 2020

Danyelza Gains Accelerated Approval to Treat Neuroblastoma

Neuroblastoma is a solid tumor of childhood that occurs in the nervous system outside of the brain. Some tumors are easily treatable, but the majority are aggressive in nature.

  • In November, the US Food and Drug Administration (FDA) approved Danyelza (naxitamab-gqgk) 40 mg/10 mL, used in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), for the treatment of patients one year of age and older with relapsed/refractory high-risk neuroblastoma in the bone or bone marrow. Use is approved under accelerated approval, and continued approval may be contingent upon confirmatory trials.
  • Danyelza, from Y-mAbs Therapeutics, is a GD2-binding monoclonal antibody. It is given as an intravenous infusion in an outpatient setting on Days 1, 3, and 5 of each treatment cycle and repeated every four weeks.
  • Danyelza contains a boxed warning for serious infusion-related reactions and neurotoxicity. Common side effects include infusion-related reaction, pain, fast heart rate, vomiting, cough, nausea, and diarrhea, among others. 

FDA OKs First-in-Class Imcivree for Rare Genetic Diseases of Obesity 

The FDA has approved Rhythm Pharmaceuticals' Imcivree (setmelanotide) for chronic weight management in patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing. These genetic variants can lead to insatiable hunger and weight gain beginning at a young age.

  • Imcivree (setmelanotide) is a melanocortin 4 (MC4) receptor agonist and is the first FDA approved therapy for these rare genetic diseases of obesity. It’s given as a subcutaneous injection once daily and titrated based on GI symptoms.
  • In open-label studies after one year of treatment, 80% of patients with obesity due to POMC or PCSK1 deficiency had greater than 10% weight loss. In those with LEPR deficiency, 45.5% of patients had greater than 10% weight loss.
  • Common side effects include injection site reaction, skin hyperpigmentation, and nausea, among others. Warnings and precautions include disturbance in sexual arousal, depression and suicidal ideation.
  • Commercial availability is expected in the first quarter of 2021.

Oxlumo Cleared as First Treatment of Primary Hyperoxaluria Type 1 

Primary Hyperoxaluria Type 1 (PH1) is a rare genetic disease that results in oxalate overproduction in the liver and can lead to the formation of kidney stones, nephrocalcinosis and deposition of oxalate in bones, eyes, skin, and heart leading to severe illness and death.

  • Oxlumo (lumasiran) is now FDA-approved for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. Oxlumo, from Alnylam Pharmaceuticals, is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1).
  • In the ILLUMINATE-A study, Oxlumo was shown to significantly reduce levels of urinary oxalate compared to placebo, with most patients reaching normal or near-normal levels. Injection site reactions were the most common side effect.
  • Oxlumo is given via subcutaneous injection once a month for three months, then once every quarter thereafter. For patients who weigh less than 10 kg, the dose is given each month.

Zokinvy Offers a Survival Benefit for Genetic Premature Aging Diseases

Progeria and progeroid laminopathies (PL) are extremely rare and distinct genetic premature aging diseases that accelerate death in young patients. Untreated children with progeria die of heart disease at an average age of 14.5 years. Currently, there are 20 children and young adults known to have these diseases in the US.

  • In response, the FDA has approved Zokinvy (lonafarnib) for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and processing-deficient Progeroid Laminopathies (PL). Zokinvy is indicated in adult and pediatric patients 12 months of age and older with a body surface area (BSA) of 0.39 m2 and above.
  • Zokinvy is a disease-modifying agent that blocks the accumulation of defective, farnesylated proteins. Data was derived from two open-label clinical trials in 62 patients. In patients with progeria, Zokinvy reduced the incidence of mortality by 60% and increased average survival time by 2.5 years.
  • Zokinvy dosing is based on body surface area (BSA) and is given as an oral capsule twice daily with morning and evening meals.
  • The most commonly reported side effects were mild or moderate gastrointestinal events (vomiting, diarrhea, nausea).

Sutab Tablets Approved as a New Option for Colonoscopy Preparation 

Colonoscopy is the most common screening method to detect colorectal cancer and is considered the gold standard.

  • In November, the FDA approved Sutab (sodium sulfate, magnesium sulfate and potassium chloride) tablets, an oral osmotic laxative preparation for colonoscopy in adults.
  • Sutab gives patients and physicians an alternative to liquid-based colonoscopy preparations. It is given as a split-dose regimen over 2-days (12 tablets in the evening and 12 tablets the next morning) with specific amounts of water. In two pivotal trials, 92% of patients achieved successful bowel cleansing with Sutab.
  • The most common gastrointestinal (stomach) side effects are nausea, abdominal distension, vomiting and upper abdominal pain. More Sutab patients reported nausea and vomiting than the comparator agents in studies.
  • Sutab, from Sebela Pharmaceuticals and marketed by Braintree, will be available on January 1, 2021.

Posted: November 2020

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