As announced, initial proof-of-concept (POC) results from distinct ongoing investigator-initiated trial (IIT) in Abu Dhabi show crofelemer reduced total parenteral support in a pediatric MVID patient with intestinal failure by up to 27%; per the IIT protocol, patient was taken off crofelemer after 12 weeks of treatment for a period of...
treatment News
CAMBRIDGE, Mass. — Biogen Inc. (Nasdaq: BIIB) – today announced that The Journal of the American Medical Association (JAMA) Neurology has published final results from the completed Phase 3 VALOR study and its open-label extension (OLE) study evaluating QALSODY® (tofersen) for the treatment of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS) with over...
BEERSE, Belgium – The Janssen Pharmaceutical Companies of Johnson & Johnson announced overall survival (OS) results from the Phase 3 MAIA (NCT02252172) study showing the addition of DARZALEX® ▼(daratumumab) to lenalidomide and dexamethasone (D-Rd) resulted in a statistically significant survival benefit over lenalidomide and dexamethasone (Rd) alone in patients with newly...
BEERSE, Belgium – The Janssen Pharmaceutical Companies of Johnson & Johnson today announced that new data for ciltacabtagene autoleucel (cilta-cel), an investigational B-cell maturation antigen (BCMA)-directed CAR-T therapy, demonstrated sustained efficacy and durable responses in heavily pretreated patients with relapsed/refractory multiple myeloma (RRMM).[1] Updated results from the Phase 1b/2 CARTITUDE-1...
BEERSE, Belgium – The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that they have submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) seeking approval of cilta-cel, an investigational B cell maturation antigen (BCMA)-directed chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of...
LEIDEN, the Netherlands – Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved Joenja® (leniolisib), an oral, selective phosphoinositide 3-kinase (PI3K) delta inhibitor, for the treatment of activated PI3K delta syndrome (APDS) in adult and...
TOKYO, Japan — Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) and Nippon Shinyaku Co., Ltd. (TOKYO: 4516) announced today that the electronic package insert for the anti-cancer agent/humanized anti-CD20 monoclonal antibody Gazyva® Intravenous Infusion 1000 mg [generic name: obinutuzumab (genetical recombination)] has been revised to allow combination therapy with the anti-cancer agent/BCL-2 inhibitor Venclexta® Tablets 10...
TOKYO & HYOGO, Japan — MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) today announced that the Ministry of Health, Labour and Welfare of Japan has granted orphan drug designation to JR-446, an investigational drug for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB or Sanfilippo syndrome...
REDWOOD CITY, Calif. – Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, today announced the initiation of a Phase 1/2 clinical trial to evaluate JSP191, the company’s anti-CD117 monoclonal antibody, as a targeted, non-toxic conditioning agent prior to allogeneic transplant in patients with GATA2-related myelodysplastic syndromes...
Red Wood City, Calif. — Jasper Therapeutics, Inc. (NASDAQ: JSPR), a biotechnology company focused on developing novel antibody therapies targeting c-Kit (CD117) to address diseases such as chronic spontaneous urticaria and lower to intermediate risk myelodysplastic syndromes (MDS) as well as novel stem cell transplant conditioning regimes, announced that the...