Somatostatin Acetate

After Europe's CHMP signed off on Moderna's combination COVID-19 and flu vaccine, CEO Stéphane Bancel praised the "EMA’s rigorous scientific review." While the FDA has flip-flopped on whether to review Moderna’s combination influenza and COVID-19 vaccine, it’s full speed ahead for the mRNA shot in Europe.The Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorization to mCombriax for people aged 50 and older. The committee's thumbs up is the first for a combo vaccine that protects against the two respiratory infections. Earlier this month, the FDA sent a refusal-to-file (RTF) letter to Moderna, explaining that the comparator arm of the successful trial backing the submission did not “reflect the best-available standard of care.” Eight days later, after meeting with Moderna, the U.S. regulator accepted the company’s application and established an Aug. 5 action date for its decision on whether to approve the shot. The hesitance of the FDA and the quick sign off from the European Medicines Agency (EMA) highlights the differing regulatory environments vaccine makers are facing in their two largest markets. The regulators were largely aligned until the installation last year of vaccine skeptic Robert F. Kennedy Jr. as the chief of Health and Human Services (HHS).“We appreciate the EMA’s rigorous scientific review,” Moderna CEO Stéphane Bancel said in a release on Friday.It’s a much-needed win for Moderna, which has slumped since its overwhelming success with mRNA vaccine Spikevax, which generated $37 billion in revenue for the company in 2021 and 2022. “The CHMP’s positive opinion represents an important milestone for respiratory virus vaccination and for Moderna, with the introduction of the world’s first flu plus COVID combination vaccine,” Bancel added. “If approved, this would be Moderna’s fourth marketed product in Europe.”  In addition to the growing anti-vaccine sentiment in the U.S.—much of it fueled by misinformation—there is particular distrust from regulators in the U.S. about mRNA products, despite evidence of their safety.In making its decision, the CHMP cited World Health Organization data, which showed there have been more than 281 million cases of COVID-19 reported in Europe up until Feb. 1 of this year. Additionally, there are up to 50 million cases of seasonal flu each year in Europe. Sleeping sickness nod The CHMP has also recommended Acoziborole Winthrop as a treatment for gambiense sleeping sickness, a parasitic disease which is transmitted by the bite of an infected tsetse fly and has killed millions in central and west Africa over the past century. The medicine was co-developed by Sanofi and the Drugs for Neglected Diseases initiative (DNDi).The single-dose oral treatment would provide a significant advance over current therapies, which require either a 10-day course of oral medicine or a combination of injections and oral therapy for advanced cases. The recommendation applies to early and advanced-stage gambiense in those ages 12 and older.“In just 20 years, we have gone from complicated treatments including arsenic derivatives with serious side effects, to today, when a single-dose, one-day therapy could safely cure patients,” Luis Pizarro, M.D., the executive director of DNDi, said in a release. “This progress is [a] testament to the transformative power of collaborative science.” Two CHMP recommendations for hives condition Meanwhile, Novartis has secured a positive opinion from the CHMP on remibrutinib as an oral treatment for adults with chronic spontaneous urticaria (CSU) who have an inadequate response to antihistamines. In September of last year, the FDA approved the Bruton’s tyrosine kinase inhibitor (BTKi) treatment for the same indication, also known as chronic hives. The medicine’s commercial moniker in the U.S. is Rhapsido.“The introduction of remibrutinib represents a major advancement … with improvements in symptoms observed as early as Week 1,” Martin Metz, M.D., of the Institute of Allergology, Charité Universitätsmedizin in Berlin, said in a release.Sanofi also gained a CHMP positive opinion for its Regeneron-partnered blockbuster, Dupixent, as a treatment for children ages two through 11 years with CSU. This is the first biologic treatment for those under age 12 with the disorder. The FDA, which approved Dupixent for CSU in April of last year, has an April decision date on whether to expand its use to those ages of two through 11.Earlier this week, the FDA signed off on Dupixent in its ninth indication, allergic fungal rhinosinusitis (AFRS). Other CHMP recommendations Ipsen has scored a CHMP positive recommendation for its Day One Biopharmaceuticals-partnered Ojemda (tovorafenib) for patients six months of age and older with relapsed or refractory pediatric low-grade glioma (pLGG), a type of cancerous brain tumor.The FDA endorsed Ojemda, a once-weekly oral therapy, in 2024. It competes with Novartis’ Tafinlar-Mekinist combo, which has a narrower label. The treatments provide an option to surgery and chemotherapy.Also scoring a CHMP nod is Mitsubishi Tanabe’s Onerji, a subcutaneous infusion of liquid levodopa and carbidopa, for adults with Parkinson’s disease. In 2015, AbbVie gained FDA approval for the combination treatment, known commercially as Duopa. The Japanese company gained Onerji in a $1.1-billion acquisition of NeuroDerm in 2017.  Crinetics has won a CHMP endorsement for Palsonify for the treatment of acromegaly, a rare hormonal disorder that usually occurs in middle-aged adults and is caused by the pituitary gland producing excess growth hormone. The FDA signed off on the selectively targeted somatostatin receptor type 2 agonist in September of last year.Also making the grade with the CHMP is X4 Pharmaceuticals’ Xolremdi for WHIM syndrome, an ultra-rare hereditary condition in which the immune system does not work properly, making patients more susceptible to bacterial and viral infections. The FDA approved the first-of-its-kind treatment in 2024. 2 CHMP rejections Vanda Pharmaceuticals has been on a roll, gaining FDA approvals for two new drugs in the last two months. But the Washington, D.C.-based drugmaker has failed to make the grade with its atypical antipsychotic drug iloperidone. The CHMP has questioned its efficacy and safety, citing that iloperidone can cause a change in the heart’s electrical activity, which can lead to a “life-threatening heart rhythm abnormality.” The risk of using iloperidone outweighs the benefits of treatment, the CHMP said.Vanda was seeking approval for iloperidone as a treatment for schizophrenia and bipolar I disorder. It has been on the market in the U.S. as Fanapt since 2009. The CHMP also swatted back Acadia Pharmaceuticals’ Rett syndrome drug Daybu, questioning its efficacy. The FDA approved the treatment in 2023.

iStock, hapabapa The regulator cited deficiencies at a third-party manufacturing facility. Camurus is seeking approval for its extended-release subcutaneous formulation of octreotide, which would allow more convenient once-monthly dosing for patients with acromegaly. The FDA has rejected Camurus’ investigational subcutaneous formulation of octreotide, dubbed CAM2029, for the treatment of the rare hormonal disorder acromegaly, the Swedish drugmaker announced on Tuesday. In a Complete Response Letter (CRL), the regulator cited “facility-related deficiencies” discovered last month during an inspection of a third-party manufacturer as the reason for the rejection and did not point to other issues, particularly as regards CAM2029’s clinical efficacy or safety data. The FDA needs to receive “satisfactory” responses from Camurus’ third-party provider before it can resolve the CRL, according to the company. CEO Fredrik Tiberg in a statement called the CRL “disappointing” but noted that the company remains “confident in the data supporting our [New Drug Application] and the potential of CAM2029 to address unmet medical needs of patients with acromegaly.” CRLs from the FDA rejecting drug candidates due to manufacturing issues are on the rise . Out of a total of 238 CRLs issued between 2017 and 2023, 30% involved quality/manufacturing issues —particularly problems with manufacturing processes, facility inspections, or chemical, manufacturing and controls. Camurus will work with its external manufacturer and with the FDA “to bring CAM2029 to patients living with acromegaly as soon as possible,” Tiberg said. To this end, the company said that it has already held labelling discussions with the regulator and that CAM2029’s prescribing information is “well advanced.” Afflicting around sixty patients per one million people, acromegaly is a rare and slowly progressive disease characterized by the abnormal growth of bones and enlarged hands, feet and facial features. Acromegaly also affects internal organs and can typically include symptoms such as headaches, joint pain and fatigue. Acromegaly is often caused by a tumor in the pituitary gland, which leads to the excess production of growth hormone and insulin growth factor-1. When uncontrolled, acromegaly worsens patients’ quality of life or can heighten their risk of death. Octreotide, the active ingredient of CAM2029, is a peptide drug that is well-known as an acromegaly therapy. It works by mimicking the function of somatostatin, a naturally occurring hormone, and activating its corresponding receptor. This mechanism of action helps suppress growth hormone, addressing the underlying cause of acromegaly. Octreotide also lowers the levels and activity of insulin and glucagon. The pharma industry has long taken advantage of octreotide’s therapeutic effects for acromegaly. Sun Pharmaceuticals won FDA approval for Bynfezia Pen (octreotide acetate) in 1988, which is indicated for the three-times-daily subcutaneous treatment of acromegaly. The drug is also approved for carcinoid tumors and vasoactive intestinal peptide tumors. Camurus’ investigational formulation is an extended-release version of octreotide that would allow for once-monthly dosing, giving patients a more convenient treatment option. In the Phase III ACROINNOVA 2 study, CAM2029 led to better biochemical control of acromegaly versus the current standard of care. Camurus’ injection also improved quality of life and symptom burden.