Rhythm Pharmaceuticals Announces that European Commission Authorized Variation for IMCIVREE® (setmelanotide) to Allow for Dosing in Patients with POMC or LEPR deficiency with Renal Impairment
BOSTON, June 22, 2022 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company committed to transforming the care of people living with rare genetic diseases of obesity, today announced that the European Commission (EC) this week authorized a variation for IMCIVREE® (setmelanotide) that allows for dosing in patients with POMC or LEPR deficiency who have mild, moderate or severe renal impairment.
IMCIVREE, a melanocortin-4 receptor (MC4R) agonist, last year received marketing authorization from the EC for the treatment of obesity and the control of hunger associated with genetically confirmed loss-of-function biallelic pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 6 years old and older.
“We are pleased the EC has recognized the impact IMCIVREE can have on the lives of patients living with hyperphagia and severe obesity caused by rare MC4R pathway diseases,” said David Meeker, M.D., Chairman, President and Chief Executive Officer of Rhythm. “The expansion of the dosing regimen to include patients with renal impairment is an important step in ensuring more patients have access to a therapy that addresses the root cause of these rare genetic diseases.”
The EC’s approval of the Company’s renal dosing variation allows for patients aged 6 years and older with POMC or LEPR deficiency and with mild, moderate, or severe renal impairment to be treated with IMCIVREE. There are no dose adjustments for patients with mild or moderate renal impairment and a separate dose titration schedule is provided for patients with severe renal impairment. Treatment is not recommended for patients with end stage renal disease.
Rhythm’s Type II variation application to the European Medicines Agency (EMA) for setmelanotide for the treatment of obesity and control of hunger in adult and pediatric patients 6 years old and older with Bardet-Biedl syndrome also is under review. The EMA’s Committee for Medicinal Products for Human Use (CHMP) is anticipated to make its recommendation on this application in the third quarter of 2022. The amendment for renal impairment dosing is being considered with the BBS application, as well.
About Rhythm Pharmaceuticals
In the EU and Great Britain, IMCIVREE is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. IMCIVREE should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology.
Rhythm’s Type II variation application to the European Medicines Agency (EMA) for the treatment of obesity and control of hyperphagia in adult and pediatric patients 6 years of age and older with BBS is under review. The Company is also continuing to advance the most comprehensive clinical research program ever initiated in MC4R pathway diseases, including the pivotal Phase 3 EMANATE clinical trial evaluating setmelanotide in four independent sub-studies in patients with obesity due to POMC insufficiency caused by heterozygous variants in the POMC or PCSK1 genes, LEPR insufficiency caused by heterozygous variants in the LEPR gene, SRC1 deficiency caused by a variant in the NCOA1 gene, and SH2B1 deficiency caused by a variant in the SH2B1 gene or 16p11.2 deletion encompassing the SH2B1 gene. The Phase 2 DAYBREAK trial is evaluating setmelanotide in patients with severe obesity and hyperphagia caused by rare variants associated with 10 prioritized MC4R-relevant genes. Rhythm has also initiated a Phase 3 pediatric trial and a Phase 3 trial evaluating a weekly formulation of setmelanotide.
IMCIVREE® (setmelanotide) Indication
Limitations of Use
WARNINGS AND PRECAUTIONS
Disturbance in Sexual Arousal: Spontaneous penile erections in males and sexual adverse reactions in females have occurred. Inform patients that these events may occur and instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention.
Depression and Suicidal Ideation: Depression and suicidal ideation have occurred. Monitor patients for new onset or worsening depression or suicidal thoughts or behaviors. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur.
Skin Pigmentation and Darkening of Pre-existing Nevi: Generalized increased skin pigmentation and darkening of pre-existing nevi have occurred. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmentary lesions.
Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants. Serious and fatal adverse reactions including “gasping syndrome” can occur in neonates and low birth weight infants treated with benzyl alcohol-preserved drugs.
USE IN SPECIFIC POPULATIONS
To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at 833-789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please see the full Prescribing Information for additional Important Safety Information.