Rhythm Pharmaceuticals Receives Positive CHMP Opinion for ▼¹ IMCIVREE® (setmelanotide) for the Treatment of Obesity and Control of Hunger in Patients with Bardet-Biedl Syndrome
-- European Commission decision anticipated in fourth quarter of 2022 --
BOSTON, July 22, 2022 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases, today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending to expand the current marketing authorization for IMCIVREE® (setmelanotide) to include the treatment of obesity and control of hunger in adult and pediatric patients 6 years of age and older with genetically confirmed Bardet-Biedl syndrome (BBS).
“The positive opinion from the CHMP marks another important step toward our goal of delivering the first precision medicine to treat severe, early-onset obesity and hyperphagia in patients living with BBS across Europe,” said David Meeker, M.D., Chairman, President and Chief Executive Officer of Rhythm. “Approximately 1,500 patients have already been diagnosed with BBS and are now being cared for at academic settings in Europe. We look forward to continuing our work with Health Technology Assessment bodies and payers across Europe to achieve access to setmelanotide for patients with BBS.”
Philip Beales, M.D., University College London, Institute of Child Health, said, “Patients living with BBS are looking for a transformational treatment that can significantly reduce hunger and body weight. If approved, setmelanotide could change the treatment paradigm for these patients and their families, providing them an option that not only has the potential to address the physical aspects of the disease, but also its negative effects on overall health, well-being and quality of life.”
The CHMP opinion will now be reviewed by the European Commission (EC), which has the authority to grant and expand marketing authorizations for medicinal products in the European Union. A final decision on the application to expand the marketing authorization for setmelanotide is anticipated in the fourth quarter of 2022.
About Rhythm Pharmaceuticals
Rhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases. Rhythm’s precision medicine, setmelanotide, is approved by the U.S. Food and Drug Administration (FDA) for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing, or patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS). The European Commission (EC) and Great Britain’s Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide 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. setmelanotide is the first-ever FDA-approved and EC- and MHRA-authorized therapy for patients living with these rare MC4R pathway diseases. Rhythm received a positive Committee for Medicinal Products for Human Use (CHMP) opinion on its Type II variation application to the European Medicines Agency seeking regulatory approval and authorization for setmelanotide to treat obesity and control of hunger in adult and pediatric patients 6 years of age and older with BBS in the European Union and a decision from the EC is expected in the fourth quarter of 2022. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity and is leveraging the Rhythm Engine and the largest known obesity DNA database -- now with approximately 45,000 sequencing samples -- to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. Rhythm’s headquarters is in Boston, MA.
Setmelanotide Indication
In Great Britain and the European Union, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed loss-of-function biallelic pro-opiomelanocortin (POMC), including PCSK1, deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 6 years of age and above.
In the United States, setmelanotide is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to POMC, PCSK1 or LEPR deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1 or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS) or Bardet-Biedl syndrome (BBS).
Limitations of Use
Setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology.
Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide would not be expected to be effective:
- Obesity due to suspected POMC, PCSK1 or LEPR deficiency with POMC, PCSK1 or LEPR variants classified as benign or likely benign
- Other types of obesity not related to POMC, PCSK1 or LEPR deficiency, or BBS, including obesity associated with other genetic syndromes and general (polygenic) obesity.
WARNINGS AND PRECAUTIONS
Skin Monitoring: Setmelanotide may lead to generalized increased skin pigmentation and darkening of pre-existing naevi because of its pharmacologic effect. Full body skin examinations should be conducted annually to monitor pre-existing and new skin pigmentary lesions before and during treatment with setmelanotide.
Heart rate and blood pressure monitoring: Heart rate and blood pressure should be monitored as part of standard clinical practice at each medical visit (at least every 6 months) for patients treated with setmelanotide.
Prolonged penile erection: Spontaneous penile erections have been reported in clinical trials with setmelanotide. Patients who have a penile erection lasting longer than 4 hours should be instructed to seek emergency medical attention for potential treatment of priapism.
Depression: In clinical trials, depression has been reported in patients treated with setmelanotide. Patients with depression should be monitored at each medical visit during treatment with setmelanotide. Consideration should be given to discontinuing setmelanotide if patients experience suicidal thoughts or behaviors.
Paediatric Population: The prescribing physician should periodically assess response to setmelanotide therapy. In growing children, the impact of weight loss on growth and maturation should be evaluated. The prescribing physician should monitor growth (height and weight) using age- and sex-appropriate growth curves.
Excipients: This medicinal product contains 10 mg benzyl alcohol in each ml. Benzyl alcohol may cause allergic reactions. Patients who are pregnant or breastfeeding should be advised of the potential risk from the excipient benzyl alcohol, which might accumulate over time and cause metabolic acidosis. This medicinal product should be used with caution in patients with hepatic or renal impairment, because of the potential risk from the excipient benzyl alcohol which might accumulate over time and cause metabolic acidosis.
ADVERSE REACTIONS
The most frequent adverse reactions are hyperpigmentation (51%), injection site reaction (39%), nausea (33%), and headache (26%).
USE IN SPECIFIC POPULATIONS
Pregnancy
There are no data from the use of setmelanotide in pregnant women. Animal studies do not indicate direct harmful effects with respect to reproductive toxicity. However, administration of setmelanotide to pregnant rabbits resulted in decreased maternal food consumption leading to embryo-foetal effects. As a precautionary measure, setmelanotide should not be started during pregnancy or while attempting to get pregnant as weight loss during pregnancy may result in fetal harm. If a patient who is taking setmelanotide has reached a stable weight and becomes pregnant, consideration should be given to maintaining setmelanotide treatment as there was no proof of teratogenicity in the nonclinical data. If a patient who is taking setmelanotide and still losing weight gets pregnant, setmelanotide should either be discontinued, or the dose reduced while monitoring for the recommended weight gain during pregnancy. The treating physician should carefully monitor weight during pregnancy in a patient taking setmelanotide.
Breast-feeding
It is unknown whether setmelanotide is excreted in human milk. A nonclinical study showed that setmelanotide is excreted in the milk of nursing rats. No quantifiable setmelanotide concentrations were detected in plasma from nursing pups. A risk to the newborn/infant cannot be excluded. A decision must be made whether to discontinue breastfeeding or to discontinue/abstain from setmelanotide therapy taking into account the benefit of breastfeeding for the child and the benefit of therapy for the mother.
Fertility
No human data on the effect of setmelanotide on fertility are available. Animal studies did not indicate harmful effects with respect to fertility.
To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337. See APPENDIX V for a list of European national reporting systems to communicate adverse reactions.
Please see the full Prescribing Information for additional Important Safety Information.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, the potential benefits of setmelanotide for patients with BBS, and our expectations surrounding potential regulatory submissions, approvals and timing thereof, including with the EC, and our business strategy and plans, including regarding commercialization of setmelanotide in Europe, the United Kingdom, the United States and other international regions. Statements using word such as “expect”, “anticipate”, “believe”, “may”, “will” and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and the other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2022 and our other filings with the U.S. Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.
Corporate Contact:
David Connolly
Head of Investor Relations and Corporate Communications
Rhythm Pharmaceuticals, Inc.
857-264-4280
dconnolly@rhythmtx.com
Investor Contact:
Hannah Deresiewicz
Stern Investor Relations, Inc.
212-362-1200
hannah.deresiewicz@sternir.com
Media Contact:
Adam Daley
Berry & Company Public Relations
212-253-8881
adaley@berrypr.com
Rachel Samuel
emotive agency
+44-208-106-7919
rachel.samuel@emotiveagency.com
____________________
1 ▼ This medicinal product is subject to additional monitoring.
To view this piece of content from www.globenewswire.com, please give your consent at the top of this page.To view this piece of content from ml.globenewswire.com, please give your consent at the top of this page.
About GlobeNewswire by notified
GlobeNewswire by notified is one of the world's largest newswire distribution networks, specializing in the delivery of corporate press releases financial disclosures and multimedia content to the media, investment community, individual investors and the general public.
Subscribe to releases from GlobeNewswire by notified
Subscribe to all the latest releases from GlobeNewswire by notified by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from GlobeNewswire by notified
Referat af ordinær generalforsamling17.4.2024 20:13:49 CEST | pressemeddelelse
Den 17. april 2024 blev der afholdt ordinær generalforsamling i Fast Ejendom Danmark A/S, CVR-nr. 28 50 09 71, hos Accura Advokatpartnerselskab, Alexandriagade 8, 2150 Nordhavn. Dagsorden 1. Valg af dirigent 2. Bestyrelsens beretning for regnskabsåret 2023 3. Fremlæggelse af årsrapport 2023 til godkendelse og forslag til resultatets anvendelse 4. Meddelelse af decharge til bestyrelsen og direktionen 5. Resultat af bestyrelsesevaluering 6. Fremlæggelse af vederlagspolitik til godkendelse 7. Præsentation af og vejledende afstemning om vederlagsrapporten 8. Godkendelse af vederlag til bestyrelsen for 2024 9. Valg af medlemmer til bestyrelsen 9a) Udvidelse af bestyrelsen fra 3 til 4 medlemmer 9b) Valg af medlemmer til bestyrelsen 10. Valg af revisor 11. Forslag fra bestyrelse eller aktionærer 11a) Bemyndigelse til bestyrelsen til at forhøje kapitalen ved kontant kapitalforhøjelse 11b) Bemyndigelse til bestyrelsen til at udstede konvertible gældsbreve 12. Bemyndigelse til generalforsamlinge
Grant of Incentive Subscription Rights in IDEX Biometrics 17 April 202417.4.2024 20:00:00 CEST | Press release
The board of directors of IDEX Biometrics ASA resolved on 17 April 2024 to issue 550,000 incentive subscription rights to employees in the IDEX Biometrics group. The grant was made under the company's 2023 incentive subscription rights plan as amended at the extraordinary general meeting on 21 December 2023. The exercise price of the subscription rights is NOK 0.15 per share and the SRs vest by 50% per year over two years. The SRs expire on 23 May 2028. Following the grant there are 18,023,725 subscription rights outstanding in IDEX Biometrics. Primary insiders IDEX discloses transactions by the following primary insider, who were granted incentive subscription rights (SRs) to IDEX shares ISIN NO0013107490: – CEO Vince Graziani was granted 250,000 SRs at NOK 0.15 per share – CTO Anthony Eaton was granted 150,000 SRs at NOK 0.15 per share – CCO Catharina Eklof was granted 150,000 SRs at NOK 0.15 per share For further information contact: Marianne Bøe, Head of Investor Relations E-mail:
Reykjavík Energy | Green Bond Auction Results17.4.2024 19:59:49 CEST | Press release
Today, April 17th, 2024, Reykjavík Energy (RE; Orkuveita Reykjavíkur) concluded a green bond auction in the green bond classes OR020934 GB and OR180255 GB. OR020934 GB bears fixed indexed interest and pays equal payments every six months with a final maturity date of September 2nd, 2034. Previously, bonds have been issued in the category with a nominal value of ISK 22,482 million. The series attracted bids totaling ISK 1,980 million nominal value at yields between 3.95-4.08%. OR accepted bids amounting to ISK 1,780 million at a yield of 4,02%. OR180255 GB bears fixed indexed interest and pays equal payments every six months with a final maturity date of February 18th, 2055. Previously, bonds in the category have been issued with a nominal value of ISK 30,521 million. The series attracted bids totaling ISK 1,315 million nominal value at yields between 3.40 -3.52%. OR accepted bids amounting to ISK 1,215 million at a yield of 3,50%. Fossar Investment Bank oversees the issuance and sale o
TGS and PGS Receive Norwegian Competition Clearance for Merger17.4.2024 19:15:00 CEST | Press release
OSLO, Norway (17 April 2024) - Reference is made to the joint stock exchange announcement on 18 September 2023 by TGS ASA ("TGS" or the "Company," OSE: TGS) and PGS ASA ("PGS," OSE: PGS) regarding the combination of the two companies (the "Merger") to establish the premier energy data company, as well as subsequent announcements on 25 October 2023 and 1 December 2023 regarding, respectively, the execution of the definitive merger agreement and approval by the extraordinary general meetings. As further announced in the update on 14 February 2024, the Merger has been subject to review by the competition authorities in the UK as well as in Norway. With respect to Norway, TGS and PGS are pleased to confirm that the Norwegian Competition Authority (the "NCA") today has provided its clearance of the Merger. With respect to the UK, the UK Competition Market Authority (the "CMA") is still in its phase I review. The deadline for the CMA to announce clearance or phase II review is 11 June 2024.
PGS and TGS receives Norwegian competition clearance for Merger17.4.2024 19:15:00 CEST | Press release
April 17, 2024, Oslo, Norway: Reference is made to the joint stock exchange announcement on 18 September 2023 by TGS ASA ("TGS" or the "Company," OSE: TGS) and PGS ASA ("PGS," OSE: PGS) regarding the combination of the two companies (the "Merger") to establish the premier energy data company, as well as subsequent announcements on 25 October 2023 and 1 December 2023 regarding, respectively, the execution of the definitive merger agreement and approval by the extraordinary general meetings. As further announced in the update on 14 February 2024, the Merger has been subject to review by the competition authorities in the UK as well as in Norway. With respect to Norway, TGS and PGS are pleased to confirm that the Norwegian Competition Authority (the "NCA") today has provided its clearance of the Merger. With respect to the UK, the UK Competition Market Authority (the "CMA") is still in its phase I review. The deadline for the CMA to announce clearance or phase II review is 11 June 2024. K