Side effects observed in testing have led Novartis to halt development of rap-cel in multiple indications and Bristol Myers to voluntarily pause its zola-cel program.

Original title: “Novartis, Bristol Myers pause autoimmune CAR-T trials due to safety concerns”

The approval of rusfertide—now to be branded Mimrylo—for polycythemia vera marks a key win for Takeda, which now has crossed the FDA finish line for two of three critical product launches slated for the coming year.

Original title: “Takeda, Protagonist score broad FDA nod in rare blood cancer, setting up $2B opportunity”

sBLA covers dMMR/MSI‑H stage II–III rectal cancer

Original title: “Jemperli accepted for priority review by US FDA”

For example, in the clinical trial that won approval for Rasonque, patients were on the drug for a median of six months. But even over several months, the costs can pile up, and some patients who respond well can stay on a cancer drug for years. Over the years, the public and lawmakers have expressed outrage at the high costs of insulin and drugs for H.I.V., hepatitis C and, most recently, obesity. One-time therapies for rare genetic diseases ... For example, in the clinical trial that won approval for Rasonque, patients were on the drug for a median of six months. But even over several months, the costs can pile up, and some patients who respond well can stay on a cancer drug for years. Over the years, the public and lawmakers have expressed outrage at the high costs of insulin and drugs for H.I.V., hepatitis C and, most recently, obesity. One-time therapies for rare genetic diseases now routinely carry price tags of a few million dollars. Rasonque, the new pancreatic cancer treatment from Revolution Medicines, illustrates the huge inflation in cancer drug prices over the past two decades. Cancer drugmakers and their defenders say the prices reflect a boom in innovation that has transformed deadly diseases into chronic ones and even produced cures. And particularly in the case of smaller companies, they say, there’s a need to recoup considerable investments in clinical trials. In a key clinical trial, patients with advanced pancreatic cancer who got the pill lived for a median of 13.2 months, compared with 6.7 months for those who got chemotherapy. Those results have electrified pancreatic cancer specialists and patients because they occurred in people who had no good treatment options and little time left to live.

Original title: “A $480,000-a-Year Pill Reflects a New Normal for Cancer Drugs - The New York Times”

Download Sample PDF Report to know ... rare metabolic disease development * Rising awareness of pancreatitis risk associated with severe hypertriglyceridemia driving demand for effective therapeutic alternatives Familial Lipoprotein Lipase Deficiency Pipeline Market Barriers * Rarity of the condition, complicating clinical trial design, ... Download Sample PDF Report to know more about Familial Lipoprotein Lipase Deficiency drugs and therapies: https://www.delveinsight.com/sample-request/familial-lipoprotein-lipase-deficiency-pipeline-insight?utm_source=openpr&utm_medium=pr&utm_campaign=kkpr Familial Lipoprotein Lipase Deficiency Pipeline Market Drivers * High unmet medical need due to the absence of approved, disease-modifying therapies beyond strict dietary management * Advances in genetic testing enabling earlier and more accurate diagnosis of LPL gene mutations * Growing research focus on antisense oligonucleotide and RNA-tar As per DelveInsight s assessment globally Familial Lipoprotein Lipase Deficiency pipeline constitutes 5 key companies continuously working towards developing 5 Familial Lipoprotein Lipase Deficiency treatment therapies analysis of Clinical Trials Therapies Mechanism of Action Route of Administration and Developments analyzes ... Press release - DelveInsight Business Research LLP - Familial Lipoprotein Lipase Deficiency Pipeline 2026: Key Companies, MOA, ROA, and Clinical Trials Evaluation by DelveInsight - published on openPR.com As per DelveInsight's assessment, globally, Oncolytic Virus Cancer Therapy pipeline constitutes 120+ key companies continuously working towards developing 125+ Oncolytic Virus Cancer Therapy treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.

Original title: “Familial Lipoprotein Lipase Deficiency Pipeline 2026: Key Companies, MOA, ROA, and Clinical Trials Evaluation by DelveInsight”

These findings support acoramidis as a therapy capable of altering the trajectory of this otherwise progressive disease,” said Marianna Fontana, M.D. of University College London, UK. “For patients and clinicians navigating ATTR-CM, this is an exciting signal that the treatment paradigm ... These findings support acoramidis as a therapy capable of altering the trajectory of this otherwise progressive disease,” said Marianna Fontana, M.D. of University College London, UK. “For patients and clinicians navigating ATTR-CM, this is an exciting signal that the treatment paradigm is shifting toward a therapy that could actively restore heart health rather than only manage decline.” These forward-looking statements include statements regarding the potential clinical significance and therapeutic implications of the data presented regarding acoramidis, including the potential clinical and therapeutic implications of observed changes in cardiac structure and function and the potential for acoramidis to alter the trajectory of ATTR-CM and restore heart health; the potential utility of AI-based tools and digital biomarkers for the detection, monitoring and screening of ATTR-CM in clinical trials and clinical practice; and BridgeBio’s plans to present additional data regarding Attruby at future medical meetings. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, initial and ongoing data from the Company’s clinical trials not being indicative of final data, the design and success of ongoing and planned clinical trials, the risk that results from post hoc analyses, subgroup analyses or other analyses may not be predictive of future clinical outcomes or treatment effects, that observed improvements in cardiac structure, function or other measures The CMR substudy of ATTRibute-CM and its open-label extension provide the first evidence from serial CMR that a therapy can potentially reverse disease progression through Month 42. The findings presented by Awais Sheikh, MBChB of the National Amyloidosis Centre, London, UK were evaluated using two complementary analytical approaches, which found: In a completer analysis, clinically meaningful improvement from baseline in left ventricular (LV) systolic function was observed in 54% of acoramidis-treated patients versus 20% of placebo-treated patients at Month 30, and in 53% of continuous-acoramidis patients at Month 42

Original title: “Acoramidis Demonstrates Reversal of Cardiac Structural Disease Progression and Functional Decline and Significantly Increases Days Alive and Free from Hospitalization in ATTR-CM - BioSpace”

Providing patients with comprehensive ... and gene therapies. Orsini partners with biopharma innovators, healthcare providers and payors to support patients and their families in accessing revolutionary treatments for rare diseases. Through integrated rare disease pharmacy solutions including pharmacy distribution, patient services, clinical management ... Providing patients with comprehensive and compassionate care since 1987, Orsini is a leader in rare diseases and gene therapies. Orsini partners with biopharma innovators, healthcare providers and payors to support patients and their families in accessing revolutionary treatments for rare diseases. Through integrated rare disease pharmacy solutions including pharmacy distribution, patient services, clinical management and convenient home infusion services, Orsini simplifies how patients connect to advanced therapies. It was tested in the VALOR study, the largest placebo-controlled dermatomyositis trial ever conducted. In VALOR, patients taking LISRAYA saw clinically meaningful improvement across a broad range of disease measures, including global myositis response, cutaneous disease, muscle strength, functional disability and steroid sparing. "We're excited to partner with Orsini in offering this potentially transformative therapy for the dermatomyositis community," Daniel Herz-Roiphe, Chief Operating Officer at Priovant, said. ELK GROVE VILLAGE, Ill., Aug. 28, 2026 /PRNewswire/ -- Orsini, a leader in rare disease pharmacy solutions, announced today that it is a specialty pharmacy partner for Priovant's LISRAYA™ (brepocitinib), a once-daily oral therapy for the treatment of adults with dermatomyositis. "This approval represents new hope for a population whose treatment options were previously very limited, and Orsini's deep experience in rare disease therapies will ensure that patients have a seamless experience as they gain access to LISRAYA."

Original title: “Orsini to Serve as Specialty Pharmacy Partner for Priovant's LISRAYA™ (brepocitinib) - BioSpace”

Shares of Germany-based BioNTech were down as much as 8% Friday after announcing a Phase 2 trial of autogene cevumeran would be discontinued for futility. The news follows Merck and Moderna’s Phase 3 win last week for their cancer vaccine plus Keytruda in melanoma.

Original title: “BioNTech halts mid-stage trial of personalized mRNA vaccine in colorectal cancer”

AstraZeneca and Ionis Pharmaceuticals’ data drop divulging more details from its highly watched Phase 3 transthyretin silencer trial “further confuses the picture” for Wainua when combined with the standard of care for ATTR-CM, analysts say.

Original title: “‘Worse than expected’ Wainua data muddies water for TTR silencers”

With the approval, Eli Lilly’s Mounjaro becomes the first dual GIP/GLP-1 receptor agonist indicated to lower the risk of heart attack, stroke or cardiovascular death in adults with type 2 diabetes.

Original title: “Lilly joins Novo in cardiovascular space after FDA nod for GLP-1/GIP agonist Mounjaro”

Lisraya’s clearance marks the first approval of a drug the Roivant subsidiary licensed from Pfizer and has since positioned against multiple rare inflammatory conditions.

Original title: “FDA green lights Priovant drug for rare skin disease”

The drug boasts a broad label, ... level of disease activity, clinical presentation or previous treatment, Priovant said. The drug can be used as both an alternative therapy or an add-on to current non-targeted DM medicines, the company explained. ... The approval was propped up by data from Priovant’s late-stage Valor trial, which the ... The drug boasts a broad label, too, cleared for use in adult DM patients without restriction based on level of disease activity, clinical presentation or previous treatment, Priovant said. The drug can be used as both an alternative therapy or an add-on to current non-targeted DM medicines, the company explained. ... The approval was propped up by data from Priovant’s late-stage Valor trial, which the company touts as the biggest DM study ever conducted. Though not without a bump along the way, Roivant-backed Priovant Therapeutics has pushed its dual TYK2/JAK1 inhibitor across the FDA finish line, securing its first approval in a rare skin disease. | Though not without a bump along the way, Roivant-backed Priovant Therapeutics has pushed its dual TYK2/JAK1 inhibitor across the FDA finish line, securing its first approval in a rare skin disease. While the company had settled on dermatomyositis as the drug’s lead indication in the pivot into phase 3, a phase 2 lupus trial flop in 2023 put a blemish on the drug’s clinical track record. Priovant is eventually hoping to score additional indications for the molecule, which is also in phase 3 testing in noninfectious uveitis and recently began enrolling patients in a late-stage study in cutaneous sarcoidosis. Roivant Pfizer autoimmune disease rare diseases U.S. In its approval announcement, Priovant noted that improvements to TIS in patients on Lisraya came as early as four weeks, increased over time and were sustained through the end of the trial period. Additionally, 55% of patients treated with Lisraya were able to achieve both moderate or better improvement on the TIS alongside minimal or no steroid use by the study’s end, as compared to 30% of patients in the control arm. In attempting to frame Lisraya’s potential merit in the DM community, Priovant highlighted patients’ own impression of their experience, noting that when asked about the overall activity of their disease, patients on Lisraya reported more than four times as much improvement as those who got the dummy drug.

Original title: “Priovant pushes ex-Pfizer asset across FDA finish line in rare skin and muscle disease | Fierce Pharma”

Through the rare disease clinic, Ginsberg helped perform several tests on Beth Synk’s daughter, Imogen “Immy” Synk, to confirm a diagnosis of Angelman syndrome, which is a rare neurogenetic condition that leads to neurodevelopmental disabilities. Synk worked with Ginsberg to help make Akron Children’s a site for phase 3 of Ultragenyx’s GTX-102 investigational antisense oligonucleotide therapy clinical trial... New York native Dr. Matthew Ginsburg shares his journey in medicine. In February, the Synks hosted a fundraiser and raised over $34,000 for the Foundation for Angelman Syndrome Therapeutics. Synk is thankful for Ginsberg. “The fact that he took the initiative and brought the trial here, it’s been tremendous for our family, but I know it will be a big deal for many families in the area,” she says. It’s families like the Synks who help inspire Ginsberg to keep pushing for rare disease treatments. “That was really a product of a family here, whose daughter has Alexander disease, which is a rare brain condition that causes sort of progressive deterioration over time,” he says. “In addition to being very instrumental in helping to fund and support the first disease-modifying therapy, they also made a charitable donation to the hospital to establish this chair and the clinic.” · An Akron Children’s patient, the Pearsons’ daughter participated in a trial at the Children’s Hospital of Philadelphia for zilganersen, an Ionis Pharmaceuticals medicine that is now before the FDA for approval. But, while at college, Ginsberg met pre-med students and worked as a respite staffer caring for a child with autism, which redirected his career pathway. “I felt like it was more important, meaningful and impactful,” Ginsberg says of medicine. He studied at the Cleveland Clinic Lerner College of Medicine at Case Western Reserve University, choosing pediatric neurology as his residency after medical school.

Original title: “Hope for the Future: Akron Children's Doctor Brings Hope for Rare Diseases - Akron Life Magazine: Akron Ohio, Restaurants and Entertainment”