Chiesi Global Rare Diseases Highlights Commitment to Rare Disease Communities at ENDO 2024

Chiesi Global Rare Diseases, a specialized division of the Chiesi Group, has recently shared significant updates from their portfolio focused on rare endocrine disorders. These updates were presented through two notable poster presentations and an oral presentation at the Endocrine Society’s ENDO 2024 Meeting, held in Boston, Massachusetts from June 1-4, 2024.

During the event, a noteworthy post hoc analysis of three Phase 3 clinical trials (CH-ACM-01, OPTIMAL, MPOWERED) highlighted the efficacy and safety of MYCAPSSA (octreotide) in treating acromegaly. Additionally, data from the Chiesi Management of Acromegaly (MACRO) registry and insights into the Lipodystrophy Severity Scoring tool were also discussed.

Giacomo Chiesi, Head of Chiesi Global Rare Diseases, expressed excitement about these developments, stating, "Our dedication to supporting individuals living with rare endocrine diseases is unwavering. With the four-year anniversary of MYCAPSSA approaching, we continue to prioritize innovative solutions for the acromegaly and lipodystrophy communities."

The oral presentation focused on the biochemical response, symptom management, and safety of oral octreotide capsules in acromegaly. Key findings from a pooled analysis of the three Phase 3 trials demonstrated a 72% response rate in patients with IGF-I values within one times the upper limit of normal. Notably, previous injectable medication doses did not significantly influence response rates. Symptom improvements included reductions in joint pain and swelling, with a median decrease of one symptom per patient and a mean decrease of 0.8 in the Acromegaly Index of Severity. The most common adverse events were gastrointestinal issues, typically occurring within two weeks and with a low discontinuation rate.

The first late-breaking poster presentation detailed real-world biochemical and symptom outcomes with oral octreotide from the MACRO registry. Among 230 patients, 32 had started MYCAPSSA at baseline, with another 26 initiating the treatment post-baseline. The mean treatment duration was 19.7 months, with most patients maintaining biochemical and symptomatic control. At the last follow-up, 87% of patients were biochemically controlled, and 71% had symptom control. The discontinuation rate was 33%, often due to insufficient biochemical or symptom control, which was sometimes linked to not increasing the dose to 80mg/day.

The second poster presentation introduced the Lipodystrophy Severity Score (LDS), a validated tool designed to assess the disease burden in lipodystrophy patients. The LDS enables comparisons across patient groups and monitors changes over time. It has shown high reliability and strong correlation with the Clinical Global Impression of severity. Future developments include an online platform to enhance its functionality and application in real-world datasets.

MYCAPSSA is indicated for the long-term maintenance treatment of acromegaly in patients who have responded to and tolerated treatment with octreotide or lanreotide. Key safety considerations include potential gallbladder issues, blood sugar, thyroid, and vitamin B12 level monitoring, and possible bradycardia or arrhythmia. Common adverse reactions include gastrointestinal disturbances, headache, joint pain, and increased blood glucose levels.

Acromegaly results from excessive growth hormone production due to a benign pituitary tumor, leading to various health issues like facial changes, joint pain, and organ enlargement. About 25,000 people in the U.S. are affected, with around 8,000 receiving injectable treatments that can cause prolonged pain and inadequate symptom control.

Lipodystrophy syndromes, which involve abnormal fat storage, are categorized into generalized and partial forms, with both inherited and acquired types. These disorders can lead to serious health complications and significantly impact quality of life.

Chiesi Global Rare Diseases continues to strive for equitable access to therapies for rare disease patients, working closely with the global community to address unmet medical needs and improve lives. The Chiesi Group, a biopharmaceutical company, is committed to developing innovative solutions in respiratory health, rare diseases, and specialty care, with a strong emphasis on social and environmental responsibility.