On October 16, 2026, Ascendis Pharma A/S announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending marketing authorization for once-weekly TransCon CNP (navepegritide). This approval targets achondroplasia treatment in children aged two years and older who have not yet reached epiphyseal closure. The final decision from the European Commission on Ascendis Pharma’s Marketing Authorisation Application (MAA) is expected in the fourth quarter of 2026.
Jan Mikkelsen, the company’s CEO, expressed optimism about the potential impact of TransCon CNP on the achondroplasia community, reinforcing its role in Ascendis's growth disorder portfolio as they aim for their Vision 2030 objectives. The positive opinion stemmed from a rigorous review of clinical data, which included three randomized, double-blind, placebo-controlled trials and data from up to three years of open-label extensions. The pivotal findings from the ApproaCH Trial are documented in publications within JAMA Pediatrics.
The EMA will now assess whether the orphan designation granted to TransCon CNP during development can be maintained as it approaches the next review phase. Should the European Commission authorize the product, Ascendis plans to launch it in Germany under the brand name YUVIWEL® in the first quarter of 2027.
On October 13, SAB Biotherapeutics, Inc. announced pivotal news, as the U.S. FDA granted Fast Track designation to SAB-142, an immunotherapy that may significantly alter the treatment landscape for individuals diagnosed with new-onset Stage 3 type 1 diabetes. This designation marks a crucial step forward in the development and expedited review of therapies aimed at addressing serious unmet medical needs.
Samuel J. Reich, SAB BIO’s CEO, highlighted the importance of this regulatory milestone, which reflects the FDA's recognition of SAB-142's potential to meet a significant therapeutic need. Currently, SAB-142 is undergoing evaluation in the registrational Phase 2b SAFEGUARD study, targeting participants diagnosed within 100 days of Stage 3 type 1 diabetes. Recruitment for this trial is anticipated to complete by the end of Q4 2026, with topline data projected for the second half of 2027. An additional Phase 3 study, the investigator-led PRISE-hATG, will further evaluate the therapy in Stage 3 type 1 diabetes patients 100 days to two years post-diagnosis.
On October 12, Insulet Corporation announced the nationwide release of Omnipod Discover™, a web-based data platform designed to enhance the diabetes management experience for Omnipod 5 users, their caregivers, and healthcare providers. This innovative platform transforms substantial diabetes data into clear visualizations and personalized insights, promoting more informed decision-making regarding diabetes therapy.
Trang Ly, MBBS, FRACP, PhD and the chief medical officer at Insulet, emphasized the growing necessity for actionable insights in diabetes management. Omnipod Discover offers users weekly reports on glucose and insulin delivery, allowing for targeted improvements in managing their condition. Furthermore, healthcare professionals benefit from an AGP that integrates key settings and medication events, streamlining clinical recommendations.
This platform has already been positively received in the U.S., with over 15,000 users and more than 1,800 healthcare professionals engaging with it during its limited release phase. Internationally, it has also reached markets in Spain, Australia, Saudi Arabia, Qatar, Kuwait, UAE, and Israel. The launch builds on evidence presented at the American Diabetes Association’s Scientific Sessions, showing increased time in range for users, thus underscoring user engagement’s vital role in effective diabetes management.
VeraMorph LLC is making strides in the development of an innovative oral formulation of fulvestrant, aimed at treating estrogen receptor positive (ER+) metastatic breast cancer (mBC). Following a recent pre-Investigational New Drug (pre-IND) meeting, VeraMorph received positive feedback from the FDA regarding a proposed clinical strategy involving studies in healthy volunteers.
The oral version of fulvestrant holds the potential for improved patient compliance and survival outcomes, particularly benefiting the approximately 70% of ER+ mBC patients who maintain a wild-type ESR1 gene. VeraMorph’s groundbreaking degradable gel technology has demonstrated significantly increased bioavailability in preclinical studies.
With funding from a recent NIH award, VeraMorph aims to complete crucial IND-enabling studies and anticipates entering the clinic with oral fulvestrant by 2027. The company plans to raise additional funds to support clinical trials and manufacturing processes related to this unique treatment approach.
On October 14, Rezolute, Inc. confirmed that their Phase 3 study, known as upLIFT, focusing on ersodetug for treating refractory hypoglycemia due to tumor-related hyperinsulinism, has completed enrollment. Topline data from this open-label study is expected to be released by mid-December 2026. The company is committed to continuing its expanded access program, which offers ersodetug to patients facing severe and resistant hypoglycemia on a compassionate use basis.