Dexcel Pharma Technologies Ltd.

TUESDAY, July 16, 2024 -- For patients experiencing migraine, direct calcitonin gene-related peptide (CGRP) inhibition with monoclonal antibodies (mAbs) is associated with reduced rates of acne and rosacea compared with no inhibition (topiramate) or indirect inhibition (triptans), according to a research letter published online July 10 in JAMA Dermatology . Christopher J. Thang, from the University of Texas Medical Branch in Galveston, and colleagues examined whether CGRP inhibition is associated with reduced rates of developing acne or rosacea in a cohort study. Patients experiencing migraines were stratified into four treatment cohorts: CGRP inhibitor (CGRPi) mAbs, CGRPi nonpeptide small molecules (gepants), triptans, and topiramate. Cohort comparisons were independently 1:1 propensity score-matched for age at index, demographics, and potential confounding medications and comorbidities using a greedy nearest-neighbor matching algorithm. The researchers found that among patients treated with CGRPi mAbs matched with those treated with triptans, CGRPi mAb exposure was associated with a significantly reduced rate of acne and rosacea. CGRPi mAb exposure was also associated with significantly reduced rates of acne and rosacea compared with topiramate. CGRPi mAb exposure was associated with a reduced rate of acne compared with gepants, although the difference was not statistically significant, and with a significantly reduced rate of rosacea. "While further studies, including randomized clinical trials, are needed to examine the effects of CGRP inhibition on acne and rosacea development, the present study highlights CGRP inhibition as a novel therapeutic avenue for both conditions," the authors write. One author disclosed receipt of personal fees from Dexcel Pharma.

ELK GROVE VILLAGE, Ill., April 18, 2024 /PRNewswire/ -- Orsini Specialty Pharmacy has been selected by Edenbridge Pharmaceuticals as the preferred specialty pharmacy partner for the distribution of Yargesa (miglustat) capsules, 100mg. YARGESA is a glucosylceramide synthase inhibitor indicated for the treatment of adult patients with Type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option. Continue Reading A rare genetic neurological condition, Gaucher disease occurs when an enzyme responsible for breaking down the lipid glucosylceramide does not work properly, resulting in the build-up of fatty substances in some organs. This can lead to enlargement and impaired function in the spleen and liver, weakened bones and problems with blood clotting. Orsini is now the preferred specialty pharmacy partner for the distribution of Yargesa (miglustat) capsules. Post this Edenbridge accepted Orsini into YARGESA's limited distribution network because of Orsini's deep experience with rare diseases, home nursing network and ability to provide comprehensive compliance programs to support patients and providers in obtaining coverage and managing therapies. "Patients with Gaucher disease are forced to live with a wide variety of symptoms, such as abdominal complaints, skeletal abnormalities, severe fatigue and seizures," Brandon Tom, Orsini's CEO, said. "We're proud to be partnering with Edenbridge to make a substantial impact on these patients' lives through treatment with YARGESA." About Orsini Specialty Pharmacy Providing patients with comprehensive and compassionate care since 1987, Orsini Specialty Pharmacy is a leader in rare diseases and gene therapies. Orsini partners with biopharma innovators, healthcare providers and payors to support patients and their families in accessing revolutionary treatments for rare diseases. Through integrated pharmacy distribution, patient services, clinical management and convenient home infusion services, Orsini delivers customized solutions that simplify how patients connect to advanced therapies. Orsini's high-touch care model centers on experienced and trained therapy care teams that provide personalized patient care to ensure that No Patient is Left Behind™. Orsini Specialty Pharmacy holds accreditations with the Accreditation Commission for Health Care (ACHC), The Joint Commission, URAC and NABP. Orsini has earned URAC's Rare Disease Pharmacy Center of Excellence Designation and ACHC's Distinction in Rare Diseases and Orphan Drugs. For more information, contact Orsini at 847-734-7373 ext. 505, email us at [email protected], or visit . SOURCE Orsini Specialty Pharmacy

The capsule is intended for patients with Type 1 Gaucher disease. Credit: Sirikarn Rinruesee/Shutterstock.com. Edenbridge Pharmaceuticals has selected PANTHERx Rare to distribute Yargesa (miglustat) capsules for the treatment of Type 1 Gaucher disease. The medication represents the first oral treatment option for adults with mild to moderate disease who are unable to use enzyme replacement therapy. The glucosylceramide synthase inhibitor Yargesa exerts its effects through the substrate reduction therapy process. This treatment method lessens the accumulation of harmful glycosphingolipids, thereby reducing symptoms. Gaucher disease is a rare genetic disorder characterised by a deficiency in the glucocerebrosidase enzyme, which leads to the build-up of a lipid known as glucosylceramide. See Also: Lonza Group gets grant for patent granted for capsule composition with pullulan, setting system, surfactant MediciNova gets grant for ibudilast higher dosage oral tablet or capsule formulations PANTHERx Rare Pharmacy CEO Rob Snyder stated: “We appreciate Edenbridge Pharmaceuticals’ commitment to making a difference in the lives of patients with Type 1 Gaucher disease. “We are looking forward to serving the needs of the patients dealing with this illness, by streamlining the process associated with getting rare medications from the people who create them to the people who need them most, ultimately delivering better health outcomes to people living with rare diseases.” In February 2024, Chiesi Global Rare Diseases, a Chiesi Group business unit, selected PANTHERx Rare for the distribution of Filsuvez topical gel. Filsuvez topical gel treats wounds associated with dystrophic as well as junctional epidermolysis bullosa in adults and paediatric patients aged six months and above.