Notizia - Recent Pharma, Healthcare and Biotech Happenings
Apr 15, 2025
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Cellenkos® Inc. announced that the FDA has granted Orphan Drug Designation to CK0801, its off-the-shelf, allogeneic T regulatory (Treg) cell therapy, for the treatment of Aplastic Anemia—a rare and life-threatening bone marrow failure disorder affecting fewer than 200,000 individuals in the U.S. The designation provides Cellenkos with regulatory and financial incentives, including potential seven-year market exclusivity upon approval. Aplastic Anemia has a projected U.S. prevalence of about 5,000 patients.
Encouraging data from a Phase I trial (NCT03773393), published in NEJM Evidence, showed CK0801 to be safe and effective, with two of three transfusion-dependent patients achieving complete transfusion independence lasting up to 3.5 years. The trial also demonstrated a 67% overall response rate and no serious adverse events, highlighting CK0801’s strong safety profile and its unique non-immunosuppressive mechanism of action.
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“Receiving Orphan Drug Designation for CK0801 in Aplastic Anemia underscores the importance of bringing novel, transformative treatment options to patients suffering from this rare disease,” said Dr. Simrit Parmar, MD, Founder of Cellenkos. Dr. Parmar is also faculty at The College of Medicine at Texas A&M University. “We aim to deliver a transformative therapeutic that can reduce the burden of blood and platelet transfusions in patients with Aplastic Anemia who have failed to respond to standard-of-care treatment.”
Beyond Aplastic Anemia, CK0801 has also shown promise in other bone marrow failure syndromes such as Myelofibrosis and Myelodysplastic Syndromes. Patients in the study experienced symptom relief, transfusion independence, and even complete remission in one case post-stem cell transplant. Cellenkos is now advancing CK0801 toward a registration trial to support regulatory approval.
Mirum Pharmaceuticals, Inc. announced that the FDA has approved a new tablet formulation of LIVMARLI (maralixibat) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC). LIVMARLI tablets are expected to be available in June through Mirum Access Plus.
“The approval of LIVMARLI in tablet form provides a meaningful additional treatment option for patients with ALGS and PFIC. It allows flexibility for patients and physicians, with the liquid dosing used by the youngest patients and a convenient one-tablet per dose option for older patients,” said Peter Radovich, president and chief operating officer at Mirum.
“We have had tremendous success with LIVMARLI since its launch, and we hope that the availability of the tablet will provide convenience that positively impacts patients’ lives,” he added.
LIVMARLI (maralixibat) is an orally administered ileal bile acid transporter (IBAT) inhibitor approved for two pediatric cholestatic liver diseases, both in liquid and tablet formulations. It is approved for the treatment of cholestatic pruritus in patients with ALGS in the U.S. from three months of age and older, and for PFIC in patients 12 months of age and older. LIVMARLI is also being evaluated in the Phase III EXPAND study in additional settings of cholestatic pruritus. For more information on ongoing clinical trials, visit Mirum’s clinical trials section on their website.
SynOx Therapeutics has received Fast Track Designation (FTD) from the FDA for emactuzumab in the treatment of Tenosynovial Giant Cell Tumours (TGCT) in patients who are not suitable for surgery or unlikely to benefit from it. Emactuzumab is a CSF-1 receptor (CSF-1R) inhibiting monoclonal antibody, currently being evaluated in the global Phase III TANGENT trial.
TGCT is a rare, non-cancerous but aggressive joint tumor that causes joint dysfunction, pain, and reduced mobility, primarily in the knees, hips, and ankles. The FTD status was supported by earlier Phase I/II data showing rapid and durable tumor reduction with a manageable safety profile. Emactuzumab has also been designated as an Orphan Medicinal Product by the EMA.
“The granting of FTD for emactuzumab in TGCT highlights the devastating toll that this disease has on patients, as well as the critical need that remains for new treatment options,” said Elyse Seltzer, M.D., Chief Medical Officer of SynOx Therapeutics.
“Based on our clinical work to date, we believe that emactuzumab has significant potential to address key patient needs by offering an effective, short-course treatment with rapid onset and a durable response that allows individuals suffering from TGCT to better manage their disease and move forward with their lives,” she added. “We look forward to completing the ongoing TANGENT study and progressing emactuzumab toward potential commercialization.”
Bristol Myers Squibb has announced that the FDA has approved OPDIVO (nivolumab) plus YERVOY (ipilimumab) as a first-line treatment for adult patients with unresectable or metastatic hepatocellular carcinoma (HCC), the most common type of primary liver cancer. The decision is based on the global Phase III CheckMate-9DW trial, which showed the combination therapy significantly improved overall survival (OS) and overall response rate (ORR) compared to lenvatinib or sorafenib monotherapy in patients who had not received prior systemic therapy.
“The CheckMate-9DW approval is an important advancement for patients, considering the incidence of liver cancer has tripled in the last four decades, yet prognosis for HCC patients remains poor,” said Aiwu Ruth He, MD, PhD, a CheckMate-9DW study investigator. “The availability of a new first-line treatment option that demonstrated a deep response can offer adults with this form of liver cancer long-term overall survival and may help address an unmet need. Given the strength of evidence from the trial, especially considering the selection and performance of a strong comparator arm, I believe that OPDIVO plus YERVOY has the potential to become a standard of care for the first-line treatment of patients with unresectable or metastatic HCC.”
In the CheckMate-9DW trial, OPDIVO plus YERVOY (n=335) achieved a median OS of 23.7 months compared to 20.6 months with lenvatinib or sorafenib (n=333), reducing the risk of death by 21% (HR=0.79; P=0.0180). The three-year OS rate was 38% with OPDIVO plus YERVOY versus 24% in the comparator arm. The combination also showed a significantly higher ORR of 36.1% vs. 13.2%, with complete responses observed in 6.9% of patients versus 1.8%, and partial responses in 29.3% vs. 11.4%. Median duration of response (mDOR) was 30.4 months with the dual immunotherapy compared to 12.9 months with the TKIs. No new safety signals were reported.
“Bringing OPDIVO plus YERVOY to patients with HCC in the first-line setting is a testament to our ongoing commitment to research and delivering important progress for people living with cancer,” said Wendy Short Bartie, senior vice president of Oncology Commercialization at Bristol Myers Squibb. “Today’s approval builds on the legacy of our dual immunotherapy and the value it has brought to patients for years. We are thrilled to add this indication for this important therapy – our second approval for OPDIVO plus YERVOY in the gastrointestinal space this week alone – and look forward to providing a new first-line treatment option to patients in need.”
OPDIVO plus YERVOY previously received accelerated approval in 2020 for second-line use in advanced HCC based on the Phase I/II CheckMate-040 trial. The new FDA decision not only converts that to a full approval but also expands its use into the first-line setting based on the CheckMate-9DW results.
Soleno Therapeutics has announced the U.S. commercial availability of VYKAT XR (diazoxide choline) extended-release tablets, the first and only FDA-approved treatment for hyperphagia in individuals with Prader-Willi syndrome (PWS) aged four years and older. The company confirmed that prescriptions of VYKAT XR have now been delivered to the first patients since its FDA approval on March 26, 2025.
“We are extremely pleased to begin delivering VYKAT XR, the only FDA-approved treatment for hyperphagia, to individuals living with PWS. Hyperphagia, the hallmark condition of PWS, is the leading cause of mortality in the PWS population and creates a significant burden for caregivers,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics. “Since FDA approval, we have seen strong interest in VYKAT XR. Our team is fully prepared to meet demand and is well-positioned to execute a successful commercial launch of this first-to-market treatment. These first shipments mark an important step in our efforts to deliver this new treatment to the PWS community and are an exciting milestone reflecting our dedication to ensuring timely access to treatment.”
Curious to learn more about the latest breakthroughs in Prader-Willi syndrome treatment?
Check out our in-depth blog: “Advances in Prader-Willi Syndrome Treatment: New Hope for Patients.”
Originally approved by the FDA in March, VYKAT XR represents a major breakthrough as the first drug approved to treat hyperphagia in PWS. The once-daily extended-release tablet acts as a potassium channel activator, targeting channels that help regulate insulin secretion, including those in the pancreas. Prader-Willi syndrome is a rare genetic condition characterized by low muscle tone, short stature, intellectual and developmental challenges, and chronic hyperphagia, which leads to life-threatening obesity if unmanaged.
Based on average weight data from clinical trials, VYKAT XR is expected to cost approximately USD 466,200 annually. Formerly known as DCCR, VYKAT XR was anticipated to become available in April 2025—and now it is.
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