Belimumab

Europe’s Committee for Medicinal Products for Human Use has recommended marketing authorization for ExCellThera’s novel cell therapy Zemcelpro in certain blood cancer patients. In a first for the bloc, Europe’s Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for a new type of stem cell transplant therapy for certain blood cancer patients. The marketing endorsement was awarded to ExCellThera’s Zemcelpro and covers a recommended approval in adults with blood cancers that require an allogeneic hematopoietic stem cell transplantation (HSCT) following myeloablative conditioning for whom no other type of suitable donor cells is available.Zemcelpro contains stem cells from a donor’s umbilical cord blood, some of which have been grown and multiplied, the European Medicines Agency (EMA) said in a press release on the decision. By boosting the number of cells, Zemcelpro makes the stem cells from a small cord blood unit more effective, the European regulator explained. For several blood cancers—including leukemias, which are detected in the blood; lymphomas, which are found in the lymph nodes; and myelodysplastic syndrome and myelomas, which are diagnosed in the bone marrow—the only current curative treatment option is allogeneic HSCT. This type of transplant involves using donated stem cells to replace the patient’s bone marrow cells to form new marrow that produces healthy blood cells. The CHMP based its Zemcelpro recommendation on pooled analysis of two single-arm trials that included 25 patients. The studies found that 84% of patients who received Zemcelpro achieved neutrophil engraftment, when donor stem cells establish themselves in the recipient’s bone marrow and produce the white blood cells known as neutrophils within a median span of 20 days.Some 10,000 new blood cancer cases requiring bone marrow transplant crop up in Europe each year, ExCellThera estimates. Many of these patients do not have access to suitable donor cells largely because of the unavailability of matched donors, the company explained. ExCellThera said it expects to gain marketing authorization from the EMA in roughly two months' time. After winning an endorsement from the CHMP, prospective medicines must go before the European Commission for a final approval verdict.ExCellThera also plans to file for approval of Zemcelpro in the U.S., U.K. and other countries. Madrigal’s Rezdiffra among others gaining CHMP nodsThe committee has also given a thumbs up to Madrigal’s Rezdiffra for the treatment of adults with noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH), a disease where fat cells accumulate in the liver causing chronic inflammation. If the drug gains marketing authorization, it would become the first treatment in the EU for the condition. Fifteen months ago, Rezdiffra became the first drug approved for MASH in the U.S. Also making the grade this month was Teva blockbuster Austedo for patients with moderate-to-severe tardive dyskinesia. The FDA approved the treatment in the indication eight years ago. Tardive dyskinesia is an irreversible movement disorder characterized by repetitive and uncontrollable movements of the tongue, lips, face, trunk and extremities and can be caused by medications used to treat mental health or gastrointestinal conditions.Meanwhile, Pfizer spinout SpringWorks Therapeutics has picked up a CHMP nod for Ogsiveo in adults with progressing desmoid tumors, which are marked by noncancerous soft tissue growths in the limbs and abdomen that often cause severe pain and disfigurement. The FDA approved Ogsiveo in the same indication in 2023.Also gaining a positive recommendation from the CHMP was Partner Therapeutics’ Imreplys for the treatment of patients exposed to myelosuppressive doses of radiation with hematopoietic sub-syndrome of acute radiation syndrome. The medicine is known as Leukine in the U.S. where it was approved in 2018. CHMP signs off on label expansions for 6 drugsAside from new approval recommendations, the CHMP has also endorsed labeled expansions for a half-dozen established medicines. The committee gave a thumbs up to Ipsen’s Cabometyx as a second-line treatment for patients with unresectable or metastatic, well differentiated extra-pancreatic (epNET) and pancreatic (pNET) neuroendocrine tumors. The FDA signed off Cabometyx in the indication three months ago. The treatment, which has been on the market since 2016, brought home sales of $1.8 billion last year.Also winning CHMP recommendations were Johnson & Johnson blockbusters Darzalex, for smoldering multiple myeloma, and Imbruvica, for patients with previously treated mantle cell lymphoma who would be eligible for autologous stem cell transplant. AbbVie partners with J&J on Imbruvica.The committee further signed off on expansions for GSK’s lupus treatment Benlysta, Bayer’s prostate cancer drug Nubeqa and Sanofi’s multiple myeloma medicine Sarclisa. CHMP opens re-examination of Lilly’s KisunlaElsewhere, fifteen months after giving a thumbs down to Eli Lilly’s Alzheimer’s disease treatment Kisunla in March of last year, the CHMP will give the medication a second look. The regulator said that it re-opened its examination of Kisunla on June 2. The FDA signed off on Kisunla in July of last year, opening up competition with Eisai and Biogen’s Alzheimer's rival Leqembi.  

Systemic sclerosis and myositis interstitial lung disease subtypes flagged as most difficult to manage; specialists cite lack of effective treatments and diagnostic clarity EXTON, PA, June 17, 2025 (GLOBE NEWSWIRE) -- According to Spherix Global Insights’ newly published Special Topix™: Interstitial Lung Disease in Rheumatology (US) report, both rheumatologists and pulmonologists report significant challenges in diagnosing and managing connective tissue disease-associated interstitial lung disease (CTD-ILD), with systemic sclerosis (SSc) and myositis (IIM) cited as the most difficult subtypes to treat. The research, based on responses from 138 US specialists (n=68 rheumatologists and 70 pulmonologists), highlights persistent unmet needs, diagnostic ambiguity, and a call for more robust and collaborative management strategies. The findings reflect that undiagnosed and misdiagnosed patients make up a substantial portion of the CTD-ILD population, with nearly one-third of rheumatologists and over half of pulmonologists naming differential diagnosis as a key challenge. Specialists underscore a lack of specific biomarkers, logistical biopsy issues, and overlapping symptoms as primary obstacles. “The most definitive test (biopsy) is invasive. It is difficult to find the clinicians who are willing to do it in a timely manner, and to convince the patient it needs to be done.” – Rheumatologist “Ensuring the correct diagnosis to avoid misdiagnosing the type of ILD or source of ILD. I usually employ the assistance of a rheumatologist.” – Pulmonologist “Often it would be difficult to get patients biopsied for logistical reasons, and often the pathology report would not be precise enough.” – Pulmonologist Even once diagnosed, CTD-ILD patients face a treatment landscape with few options to slow or halt disease progression. When asked about the most difficult aspects of management, specialists consistently pointed to a lack of effective and targeted therapies, with SSc-ILD and myositis-ILD receiving the highest unmet need scores. For SSc-ILD specifically, not only is it associated with the most aggressive progression and worst long-term outcomes, but it also presents a uniquely complex therapeutic challenge. The clinical burden is especially pronounced in patients with diffuse cutaneous systemic sclerosis (dcSSc), where the use of corticosteroids is often limited due to the heightened risk of scleroderma renal crisis. This leaves physicians with a narrow band of anti-fibrotic and immunosuppressive options, many of which are perceived as only modestly effective in controlling ILD progression, let alone reversing it. Even therapies with regulatory approval for SSc-ILD, such as Boehringer Ingelheim’s OFEV (nintedanib), garner only measured enthusiasm from treating physicians. While rheumatologists acknowledge nintedanib’s potential to slow functional decline, they note it does little to address the underlying autoimmune pathology. Similarly, mycophenolate mofetil and rituximab remain staples of clinical practice, yet their real-world performance is viewed as inconsistent and difficult to monitor. Across the board, satisfaction ratings for currently utilized agents fall short of expectations, reinforcing a treatment landscape still dominated by trial-and-error approaches. This disconnect between disease complexity and treatment efficacy is fueling demand for dual-acting agents, therapies that can address both systemic autoimmunity and pulmonary fibrosis. Nowhere is this demand more visible than in systemic lupus erythematosus-associated ILD (SLE-ILD), where rheumatologists express growing interest in GSK’s Benlysta (belimumab). Though not currently approved for ILD, belimumab’s immunologic mechanism and expanded label for lupus nephritis have raised hopes that similar benefit may extend to pulmonary manifestations. Across interviews and survey feedback, physicians articulate a desire for treatments that reduce systemic activity while simultaneously preventing fibrotic progression—without the need for an antifibrotic add-on with limited synergy. Looking ahead, specialists are cautiously optimistic about the pipeline. While interest in agents with antifibrotic and immunomodulatory properties is rising, physicians emphasize that what’s most needed is not just another drug, but a paradigm shift—a treatment model that reflects the multifaceted nature of CTD-ILD. As one physician summarized, the goal is to "treat the patient, not just the lungs." In addition to tracking physician sentiment and practice patterns, the study includes target product profile evaluations for several pipeline therapies: Benlysta (GSK), BMS-986278 (Bristol Myers Squibb), Nerandomilast (Boehringer Ingelheim), and Treprostinil (United Therapeutics). These profiles offer manufacturers timely insights into clinical expectations, adoption potential, and differentiation opportunities in an increasingly complex ILD treatment landscape. Special Topix ™ is an independent service that includes access to a report or series of reports based on current events or topics of interest in specialty markets covered by Spherix. About Spherix Global Insights Spherix is a leading independent market intelligence and advisory firm that delivers commercial value to the global life sciences industry, across the brand lifecycle. The seasoned team of Spherix experts provides an unbiased and holistic view of the landscape within rapidly evolving specialty markets, including dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology, and hematology. Spherix clients stay ahead of the curve with the perspective of the extensive Spherix Physician Community. As a trusted advisor and industry thought leader, Spherix’s unparalleled market insights and advisory services empower clients to make better decisions and unlock opportunities for growth. To learn more about Spherix Global Insights, visit spherixglobalinsights.com or connect through LinkedIn . For more details on Spherix’s primary market research reports and interactive dashboard offerings, visit or register here: https://clientportal.spherixglobalinsights.com NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight’s analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release. Andy Stankus, Rheumatology Franchise Head Spherix Global Insights 4848794284 andy.stankus@spherixglobalinsights.com Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.