Archive for category: News

The FDA has approved Palsonify™ (paltusotine) for adults with acromegaly who did not respond adequately to surgery or are not candidates for surgical treatment.

PALSONIFY, a selectively-targeted somatostatin receptor type 2 nonpeptide (SST2) agonist, is now the first once-daily, oral treatment approved for adults with acromegaly.

“With the FDA approval of our lead therapy Palsonify, today marks a new era for those living with acromegaly and also for Crinetics as a company,” said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. “We are very pleased to be fulfilling our commitment to transforming patient lives. This approval is the first to come from our deep pipeline of first-in-class, small molecule drugs. This would not be possible without the help and partnership of people living with acromegaly, their caretakers, our employees, and the clinical researchers and health care professionals who contributed to Palsonify’s successful development program. Thank you to all involved.”

The approval is based on data from the PATHFNDR-1 and PATHFNDR-2 Phase 3 pivotal trials, which evaluated PALSONIFY’s safety and efficacy in previously treated and medically untreated adults with acromegaly. Across both trials, PALSONIFY consistently demonstrated rapid onset, reliable biochemical control, and sustained efficacy.

Participants also reported significant reductions in signs and symptoms associated with acromegaly as measured by the Acromegaly Symptom Diary (ASD) — an FDA-aligned patient-reported outcome tool developed to capture the symptoms that matter to people living with acromegaly. Symptoms include headaches, joint pain, sweating, fatigue, weakness, swelling, and/or numbness/tingling. PALSONIFY was generally well-tolerated, with no serious adverse events reported in the randomized controlled portion of the trials.

Long-term results from the open-label extension (OLE) phases of both trials were presented at this year’s Endocrine Society’s annual meeting, ENDO 2025, providing further evidence of PALSONIFY’s ability to deliver durable IGF-1 control, sustained improvements in patient symptom burden, and a consistent safety profile. Ninety-one percent of patients from PATHFNDR-1 and 97 percent of completers from PATHFNDR-2 enrolled in the OLE.

“The PATHFNDR clinical development program set a new standard for acromegaly treatment by demonstrating the ability of Palsonify to drive both biochemical and symptom control, regardless of the degree of underlying disease severity,” said Dr. Shlomo Melmed, Executive Vice President of Medicine and Health Sciences and Dean of the Medical Faculty at Cedars-Sinai. “The approval of Palsonify is a significant advancement for our patients, as there is an unmet need for an easy-to-administer and safe therapeutic option with a rapid action and durable response that can consistently manage acromegaly.”

“For people living with acromegaly, treatment once meant burdensome injections, breakthrough symptoms, and lifestyle sacrifices just to stay on track,” said Jill Sisco, President of Acromegaly Community. “What matters most to our community – maintaining consistent control so the disease doesn’t control us – led us to partner with the FDA on Externally Led Patient-Focused Drug Development meetings. This new treatment reflects that our voices have been heard in shaping the next generation of acromegaly care.”

PALSONIFY is expected to be available in the U.S. in early October. Crinetics is ensuring broad access to PALSONIFY by working closely with payers, healthcare providers, and patient advocacy organizations to support those who may benefit from this treatment.

As part of this commitment, Crinetics has launched CrinetiCARE®, a comprehensive support program designed to assist people living with acromegaly throughout their treatment journey. CrinetiCARE provides disease and product education, benefit verification, financial assistance resources, and access to dedicated nurse educators who can offer support with treatment onboarding and ongoing adherence. 

A Marketing Authorization Application (MAA) for paltusotine in acromegaly is currently under review for use in the European Union, and the current timeline for the Committee for Medicinal Products and Human Use (CHMP) opinion is the first half of 2026. Crinetics is in partnership with Sanwa Kagaku Kenkyuso (SKK) to develop and commercialize paltusotine for acromegaly in Japan.

Paltusotine is also being evaluated for the treatment of carcinoid syndrome in the pivotal Phase 3 CAREFNDR trial.

For many older women, the question of whether to continue breast cancer screening has been uncertain. While most guidelines recommend mammograms up to age 74, advice for women 75 and older has been less clear. Now, a new study from researchers at the UCLA Health Jonsson Comprehensive Cancer Center suggests that regular mammograms may still offer significant benefits for women in their 80s.

The study, published in the Annals of Surgical Oncology, found that women in their 80s who get regular mammograms are more likely to have breast cancer detected early, need less aggressive treatment and live longer.

“When cancer is found on screening, it is often early stage,” said Dr. Nimmi Kapoor, an associate professor of surgery at the David Geffen School of Medicine at UCLA and senior author of the study. “In postmenopausal women with the most common hormone-sensitive breast cancers, we can often omit sentinel lymph node biopsy, chemotherapy, and sometimes even radiation. Screening is especially important in this era of de-escalation because early detection allows us to safely reduce the intensity of treatment while still achieving excellent outcomes.”

The number of older adults in the U.S. has grown rapidly over the past decade, and age is a major risk factor for breast cancer. As a result, determining the most effective way to screen older women has become increasingly important, the researchers noted. Yet guidance is limited, leaving many women and their doctors uncertain about when to continue or stop routine mammograms. Screening in older women also raises concerns about overdiagnosis, when cancers are detected that may never cause problems, and the associated costs.

With limited data on the benefits of mammography for women over 80, the researchers set out to compare outcomes between those who continued regular screening and those who did not.

The team analyzed medical records of 174 women aged 80 and older who were diagnosed with breast cancer at UCLA between 2013 and 2020. Most cancers were estrogen receptor–positive and HER2-negative and were mostly stage 1 or 2. Patients were divided into two groups: Those who had a mammogram within two years before their diagnosis (98 women) and those who did not (76 women). They then compared outcomes —including cancer stage at diagnosis, treatment intensity, and overall survival— between the two groups.

They found that women who did not have regular mammograms were more likely to have tumors that were advanced, high-grade or noticeable by touch. Screened women were more likely to have surgery to remove the tumor, while unscreened women sometimes omitted surgery altogether.

After a median follow-up of 55 months, they found women who were screened had a 55% lower risk of their cancer returning and a 74% lower risk of dying compared with women who were not screened. These advantages remained even after accounting for age, tumor type, and whether they had surgery.

“We were surprised to see such a significant survival difference among these women in their 80s,” said Kapoor, who is also an investigator in the UCLA Health Jonsson Comprehensive Cancer Center. “Our findings underscore the importance of encouraging breast cancer screening in elderly patients, regardless of age, unless they have more pressing health issues. Current guidelines are vague and often left to the provider’s discretion, so studies like ours help provide much-needed data for this underrepresented population.”

While the findings are encouraging, the researchers noted some limitations. The study looked back at medical records and only included women who were ultimately diagnosed with breast cancer, so it doesn’t capture potential downsides of screening, such as false positives, extra tests, or the emotional and financial stress on patients and their families. Larger studies are needed to confirm the benefits of mammograms for older women and to guide clearer screening recommendations.

Reference:

Siu‑Yuan Huang, Impact of Screening Mammography on Breast Cancer Outcomesin Women Aged 80 Years and Over, Annals of Surgical Oncology, https://doi.org/10.1245/s10434-025-18288-4