Craniopharyngioma

Rhythm Pharmaceuticals Announces Clinical Development Plan of Setmelanotide for Hypothalamic Obesity in Japan

Retrieved on: 
Thursday, February 22, 2024
Incidence, EMA, MC4R, Obesity, FDA, Growth, Astrocytoma, Polyphagia, Prevalence, Hunger, PMDA, European Medicines Agency, Weight gain, Patient, Brain, Craniopharyngioma, Pharmaceutical industry

BOSTON, Feb. 22, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today announced plans to add a cohort of patients with hypothalamic obesity in Japan to its ongoing global Phase 3 clinical trial of setmelanotide, with dosing expected to begin in the third quarter of 2024.

Key Points: 
  • “Following constructive discussions with Japan’s Pharmaceuticals and Medical Devices Agency (PMDA), we are pleased to have developed a clear and efficient plan to support the potential approval of setmelanotide for hypothalamic obesity in Japan,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm.
  • The condition most frequently follows the growth and surgical removal or other treatment of craniopharyngioma, astrocytoma, or other rare brain tumors.
  • These individuals often experience rapid weight gain, a reduction in energy expenditure, and an increase in hunger leading to severe obesity within six to 12 months following the hypothalamic lesions.
  • Rhythm also announced today that it completed enrollment in the pivotal, 120-patient cohort in its global Phase 3 trial of setmelanotide in hypothalamic obesity with patients, aged 4 years or older with hypothalamic obesity randomized 2:1 to setmelanotide therapy or placebo for a total of 60 weeks, including up to eight weeks for dose titration.

Rhythm Pharmaceuticals Receives Orphan Drug Designation from European Medicines Agency for Setmelanotide for Treatment of Acquired Hypothalamic Obesity

Retrieved on: 
Tuesday, September 19, 2023
Polyphagia, Brain, Obesity, International, European Medicines Agency, Rhythm, Weight gain, Astrocytoma, EMA, Patient, Medicine, European, MC4R, Diagnosis, Growth, United, Hunger, Craniopharyngioma, Disease, Pharmaceutical industry

“Acquired hypothalamic obesity is a serious disease with severe implications for patients and families and no effective treatment options,” said Yann Mazabraud, Executive Vice President and Head of International at Rhythm.

Key Points: 
  • “Acquired hypothalamic obesity is a serious disease with severe implications for patients and families and no effective treatment options,” said Yann Mazabraud, Executive Vice President and Head of International at Rhythm.
  • Rhythm is evaluating setmelanotide in a global Phase 3 clinical trial in acquired hypothalamic obesity and expects to complete patient enrollment in the fourth quarter of 2023.
  • Acquired hypothalamic obesity is a rare form of extreme obesity that occurs following damage to the hypothalamic region of the brain, which includes the MC4R pathway and is responsible for controlling physiological functions such as hunger and weight regulation.
  • Rhythm estimates there are approximately 3,500 to 10,000 patients living with acquired hypothalamic obesity in the European countries of Germany, France, Spain, Italy, The Netherlands, and the United Kingdom.1

Rhythm Pharmaceuticals Announces Pre-Marketing Early Access Authorization for Setmelanotide for Use in Patients with Hypothalamic Obesity in France

Retrieved on: 
Monday, August 7, 2023
Polyphagia, Brain, Obesity, International, Setmelanotide, Prevalence, National Agency for Food and Drug Administration and Control, Weight loss, Rhythm, Bardet–Biedl syndrome, Medication, Palestinian National Authority, Appetite, Weight gain, Teaching hospital, Astrocytoma, BBS, Incidence, HAS, Patient, ANSM, Growth, Body mass index, AP1, Hunger, Safety, Craniopharyngioma, Pharmaceutical industry, Nutrition, MC4R, BMI

BOSTON, Aug. 07, 2023 (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 French National Agency for Medicines and Health Products Safety (ANSM) and French National Authority for Health (HAS) have granted pre-marketing early access authorization AP1 (Autorisation d’Accès Précoce), for IMCIVREE® (setmelanotide), an MC4R agonist, for patients with lesional hypothalamic obesity.

Key Points: 
  • Setmelanotide has been available through a similar program for patients in France with Bardet-Biedl syndrome (BBS) since July 2022.
  • Rhythm estimates there are approximately 500-2,000 patients living with hypothalamic obesity in France, with approximately 50 to 200 new cases each year.
  • In the United States, the Company estimates the prevalence in hypothalamic obesity to be approximately 5,000-10,000 patients with an annual incidence of approximately 500 new patients.
  • “This AP1 addresses a significant unmet need for patients with severe obesity and hyperphagia, two hallmark symptoms of hypothalamic obesity, for the first time.”

St. Jude Children’s Research Hospital Celebrates 60th Anniversary and 19 Years of the St. Jude Thanks and Giving Campaign With a Week of Stories On NBC’s TODAY Show

Retrieved on: 
Thursday, November 17, 2022
TV and Radio, Managed Care, Foundation, Philanthropy, Hospitals, Science, Family, Consumer, Entertainment, Health, Other Philanthropy, Research, Teens, Parenting, Children, Baby, Maternity, Oncology, Father, Ewing's sarcoma, Friends, Food, The Plaza, Cancer, Insurance, Love, National Cancer Registrars Association, Life, Housing, Survivor, Town square, NCI-designated Cancer Center, Child, Craniopharyngioma, Travel, Hospital, National Cancer Institute, Hope, The Holocaust and the United Nations Outreach Programme, Survival rate, Patient, Fashion, Jude the Apostle

We are so grateful to our St. Jude Thanks and Giving partners and friends at TODAY for helping highlight our patients journeys and showing the difference that every person can make when they give to St. Jude.

Key Points: 
  • We are so grateful to our St. Jude Thanks and Giving partners and friends at TODAY for helping highlight our patients journeys and showing the difference that every person can make when they give to St. Jude.
  • St. Jude Childrens Research Hospital is leading the way the world understands, treats and defeats childhood cancer and other life-threatening diseases.
  • Visit St. Jude Inspire to discover powerful St. Jude stories of hope, strength, love and kindness.
  • Support the St. Jude mission by donating at stjude.org , liking St. Jude on Facebook , following St. Jude on Twitter , Instagram , LinkedIn and TikTok , and subscribing to its YouTube channel.

Rhythm Pharmaceuticals Presents Data from Phase 2 and Long-term Extension Trials Evaluating Setmelanotide for the Treatment of Hypothalamic Obesity at ObesityWeek® and Plans to Initiate Phase 3 Trial in Early 2023

Retrieved on: 
Wednesday, November 2, 2022
Body mass index, Degenerative disease, Anti-obesity medication, MC4R, Obesity, GLOBE, Appetite, Patient, Division, BMI, Nasdaq, Department, Breakthrough therapy, Craniopharyngioma, Polyphagia, University, FDA, Human, Radiation, University of Washington, Pharmaceutical industry, Medical imaging, Endocrinology

BOSTON, Nov. 02, 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 positive Phase 2 results and long-term extension trial data in hypothalamic obesity that are being presented today as a late-breaking poster at The Obesity Society’s ObesityWeek® 2022 conference in San Diego.

Key Points: 
  • As announced this week , the FDA granted Breakthrough Therapy designation to setmelanotide for the treatment of hypothalamic obesity.
  • Consistent with prior clinical experience in other rare MC4R pathway diseases, setmelanotide was observed to be generally well tolerated.
  • The ObesityWeek poster also included data on patients who continued on setmelanotide therapy in Rhythms open-label, long-term extension trial after completing 16 weeks on setmelanotide therapy in the Phase 2 trial.
  • Setmelanotide has shown significant potential to transform the care of individuals living with the onset of extreme weight gain from hypothalamic obesity.

Brain Cancer Diagnostics Market Report 2022: Increase in Brain Cancer and Cigarette Smokers Fueling Growth - ResearchAndMarkets.com

Retrieved on: 
Tuesday, September 13, 2022
Health, Oncology, Medical Devices, Incidence, COVID-19, Brain, Prevalence, Cancer Research UK, Glioblastoma, CM3, Neoplasm, Meningioma, MRI, SES Platform Services, Growth, American Association, Nuclear magnetic resonance spectroscopy, Ganglioneuroma, PET, Cancer, American Association of Neurological Surgeons, Solution, CT, Ependymoma, Congress of Neurological Surgeons, Craniopharyngioma, Cancer Australia, CNS, H. Lee Moffitt Cancer Center & Research Institute, Astrocytoma, Ageing, Association, EEG, Hospital, Oligodendroglioma, Biopsy, Electroencephalography, MRS, CAGR, Medical imaging

The global brain cancer diagnostics market is expected to grow from US$ 1,639.10 million in 2022 to US$ 7,378.57 million by 2028.

Key Points: 
  • The global brain cancer diagnostics market is expected to grow from US$ 1,639.10 million in 2022 to US$ 7,378.57 million by 2028.
  • The increase in brain cancer worldwide and the rise in cigarette smokers boost the growth of the global brain cancer diagnostics market.
  • Thus, the increase in the prevalence of brain cancer worldwide highly demands brain cancer diagnostic tools.
  • Based on cancer type, the brain cancer diagnostics market is segmented into acoustic neuroma, astrocytomas, craniopharyngiomas, ganglioneuromas, glioblastoma multiforme, ganglioneuroma, meningiomas, ependymomas, oligodendroglioma, low-grade tumors, and others.

Global Genes Awards 2022 Health Equity Grants to Rare Disease Groups Addressing Challenges Affecting Underserved Communities

Retrieved on: 
Tuesday, July 19, 2022
Health, Science, Other Science, Research, Clinical Trials, Research, CEO, Disease, Patient, Clingen, Foundation, Global Genes, Cystic fibrosis, Asia-Pacific, Program, Love, Hindi, Goal, Hope, Translation, PFIC, Caregiver, XLH, Organization, Barbershop Quartet (health outreach program), Horizon Therapeutics, Color, PWS, Diagnosis, International, CF, Therapy, Craniopharyngioma, Education, Awareness, Population, Pharmaceutical industry, 3D printing

Global Genes , a leading rare disease patient advocacy organization, today announced the recipients of its annual Health Equity RARE Patient Impact Grant .

Key Points: 
  • Global Genes , a leading rare disease patient advocacy organization, today announced the recipients of its annual Health Equity RARE Patient Impact Grant .
  • For individuals and families facing a rare disease, the journey is already incredibly difficult, said Craig Martin, CEO of Global Genes.
  • For those in historically underserved or marginalized communities, the struggle is often even more challenging.
  • If you or someone you love has a rare disease or are searching for a diagnosis, contact Global Genes at 949-248-RARE or visit our Resource Hub .

Accuray CyberKnife® System Real World Data and Clinical Studies Show Benefits in Treatment of Neurological Indications

Retrieved on: 
Tuesday, July 12, 2022
Movement, SEC, Radiosurgery, Security (finance), Neoplasm, U.S. Securities and Exchange Commission, Radiation, Craniopharyngioma, YouTube, LinkedIn, Congress, Risk, Patient, Neurosurgery, Brain, COVID-19, Cyberknife, Private Securities Litigation Reform Act, Synchrony, Time, Investment, Health, Forward-looking statement, Toxicity, Research, Head, Twitter, Industry, ISRS, NASDAQ, 2021 Essex County Council election, Medical imaging, Pharmaceutical industry, Facebook

Through investments in research and development and industry partnerships we will continue to introduce best-in-class innovations that make an incredible system even better."

Key Points: 
  • Through investments in research and development and industry partnerships we will continue to introduce best-in-class innovations that make an incredible system even better."
  • The CyberKnife System delivers frameless radiosurgery treatments, unlike other systems that use a fixed metal frame attached to the patient's head to prevent movement during treatment.
  • The robot maneuvers around the patient to deliver multiple beams of radiation from thousands of angles to target the tumor or lesion.
  • Accuray is committed to expanding the powerful potential of radiation therapy to improve as many lives as possible.

Gold House, other API brands to usher in Year of the Tiger with campaign for St. Jude Children's Research Hospital

Retrieved on: 
Thursday, January 27, 2022
Lunar New Year, View, Fanconi anemia, Survival, Travel, Qi, Awareness, World, Patient, Book, Courage, St. Jude Children's Research Hospital, TikTok, Gold, Craniopharyngioma, Hope, Child, NCI-designated Cancer Center, Twitter, Multimedia, National Cancer Institute, Research, Cancer, Food, Drink, The Gold Houses, Tiger, Beauty, Retinoblastoma, API, Hospital, LinkedIn, Eye, Love, World Health Organization, Housing, Condiment, Sale, The Year of the Tiger, Birth control, Instagram, Jude the Apostle, Facebook

The public and partnerships like this one make it possible for St. Jude to save children together.

Key Points: 
  • The public and partnerships like this one make it possible for St. Jude to save children together.
  • A limited-edition Tiger plush toy named after St. Jude patient Maelin-Kate is available via the St. Jude Gift Shop .
  • St. Jude Children's Research Hospital is leading the way the world understands, treats and defeats childhood cancer and other life-threatening diseases.
  • Join the St. Jude mission by visiting stjude.org , liking St. Jude on Facebook , following St. Jude on Twitter , Instagram and TikTok , and subscribing to its YouTube channel.

Saniona Initiates Phase 2b Clinical Trial of Tesomet for Hypothalamic Obesity

Retrieved on: 
Wednesday, November 17, 2021
Hypothalamus, Population, Mortality, Degenerative disease, Raymond, Non-alcoholic fatty liver disease, Type 2 diabetes, Disorder, Hypertension, Hunger, Type, Clinical trial, Depression, Prader–Willi syndrome, Lethargy, United Kingdom, Diabetes, Weight gain, Survivor, Disease, Human, CP, Heart, HO, Body mass index, Stroke, Amnesia, Somnolence, Polyphagia, Database, Patient, Metoprolol, Awareness, Vanderbilt University Medical Center, Food, OMX, Drug discovery, Attention deficit, Waist, Assistant, PWS, Craniopharyngioma, Pediatric endocrinology, Risk, FDA, Child, Brain, Obesity, Pharmaceutical industry

Saniona (OMX: SANION), a clinical stage biopharmaceutical company focused on rare diseases, today announced the initiation of a Phase 2b clinical trial of Tesomet in patients with hypothalamic obesity (HO).

Key Points: 
  • Saniona (OMX: SANION), a clinical stage biopharmaceutical company focused on rare diseases, today announced the initiation of a Phase 2b clinical trial of Tesomet in patients with hypothalamic obesity (HO).
  • The initiation of this Phase 2b clinical trial represents the culmination of the significant work and combined expertise of our clinical, regulatory, technical operations and quality teams, said Rudolf Baumgartner, M.D., Chief Medical Officer and Head of Clinical Development at Saniona.
  • We are also preparing to initiate a Phase 2b clinical trial of Tesomet in Prader-Willi syndrome before the end of the year, which, combined with our ongoing Phase 1 trial for SAN711, means our team will end the year with three rigorous clinical programs underway.
  • The Phase 2b clinical trial includes a randomized, double-blind, placebo-controlled 36-week treatment period followed by a 36-week open-label extension period.