Orphan Drug Designation
Nov 21, 2023
Key Updates on Phase 1 Trial of AB-1005 Gene Therapy for Multiple System Atrophy-Parkinsonian Type; European Commission Approves EBGLYSS; Bayer Stopped OCEANIC-AF Study; Pfizer and Astellas’ XTANDI Approved by FDA; FDA Orphan Drug Designation to Epic Bio’s EPI-321; FDA Fast Track Designation to Chemomab’s CM-101 for PSC
AskBio Announces First Patient Randomized in Phase 1 Trial of AB-1005 Gene Therapy for Multiple System Atrophy-Parkinsonian Type Asklepios BioPharmaceutical, Inc., a gene therapy firm fully owned and independently operated under Bayer AG, announced the initiation of the Phase I REGENERATE MSA-101 clinical trial ...
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Nov 14, 2023
Takeda’s ADZYNMA Approved by FDA; AskBio Presents Preliminary Data from Phase I Trial of Gene Therapy for CHF; Bayer’s Aflibercept 8 mg Recommended for Approval in EU; FDA Orphan Drug Designation to MAIA Biotechnology’s THIO; First Patient Dosed in Phase III ProstACT GLOBAL Study; FDA Grants Orphan Designation for Lipella’s LP-310 Drug Candidate
Takeda’s ADZYNMA Approved by FDA as the First and Only Recombinant ADAMTS13 Enzyme Replacement Therapy for the Treatment of Congenital Thrombotic Thrombocytopenic Purpura Takeda has received FDA approval for ADZYNMA (ADAMTS13, recombinant-krhn) for both prophylactic and on-demand treatment in adults and pediatri...
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Nov 07, 2023
New Asundexian Phase III Study Result; Zibotentan/Dapagliflozin Combination Demonstrated Significant Albuminuria Reduction Chronic Kidney Disease; Orphan Drug Designation to Rhenium Obisbemeda; FDA Approves Merck’s KEYTRUDA Plus Gemcitabine and Cisplatin for Biliary Tract Cancer; Orphan Drug Designation to Ayala’s AL102; GARDP Announces Successful Phase 3 Trial of of Uncomplicated Gonorrhea
Positive Results Announced in Largest Pivotal Phase 3 Trial of a First-in-Class Oral Antibiotic to Treat Uncomplicated Gonorrhea The Global Antibiotic Research & Development Partnership (GARDP), in partnership with Innoviva, Inc. (Nasdaq: INVA), had announced a significant milestone. They revealed that zolif...
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Oct 24, 2023
FDA Approves PENBRAYA for Most Common Serogroups Causing Meningococcal Disease; BIMZELX Approved Moderate to Severe Plaque Psoriasis; FDA Approves BioMarin’s VOXZOGO; FDA Fast Track Designation to ANPD001 for Parkinson’s Disease; UCB Announces FDA Approval of ZILBRYSQ; EMA Granted Orphan Drug Designation to Lisata’s LSTA1
UCB announces FDA approval of ZILBRYSQ for the Treatment of Adults with Generalized Myasthenia Gravis On the 17th of October 2023, UCB (Euronext Brussels: UCB) made an announcement regarding the approval of ZILBRYSQ® (zilucoplan) by the US FDA for the management of generalized myasthenia gravis (gMG) in adult pa...
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Oct 17, 2023
Novo Nordisk to Acquire Ocedurenone; FDA Awards Orphan Drug Designation to SLS009 in AML; FDA Approves Adjuvant Nivolumab in Completely Resected Stage IIB/C Melanoma; Fast Track Designation to South Rampart Pharma’s SRP-001; TAGRISSO + Chemotherapy Granted Priority Review in the US; Fast Track Designation to SurVaxM for Glioblastoma
South Rampart Pharma Receives U.S. FDA Fast Track Designation for SRP-001 for Acute Pain On October 12, 2023, South Rampart Pharma, Inc. announced that the U.S. Food and Drug Administration (FDA) had granted Fast Track status to its drug candidate, SRP-001, intended for the management of acute pain. SRP-001 is a...
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Oct 10, 2023
Amgen to Acquire Horizon Therapeutics; Sanofi and Teva Announce Collaboration; Boehringer Obesity Drug Trial Update; FDA Places Partial Clinical Hold on IND for Lacutamab in CTCL/PTCL; Anaptys Announces Phase 3 Clinical Trial Results of Imsidolimab; Orphan Drug Designation to GC Biopharma’s GC1126A
Sanofi and Teva Announce Exclusive Collaboration to Deliver Inflammatory Bowel Disease Treatment Sanofi and Teva Pharmaceuticals, a subsidiary of Teva Pharmaceutical Industries Ltd. in the United States, have announced a collaboration to co-develop and co-commercialize asset TEV'574, which is currently in Phase ...
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Oct 03, 2023
AbbVie Presents Phase III CANOVA Study Results; Novartis’ Iptacopan Shows Promise in Phase III Study; Fast Track Designation to AVB-001 for R/R Platinum-Resistant Ovarian Cancer; FDA Issues Complete Response Letter for Lebrikizumab; Nedosiran Approveed for Primary Hyperoxaluria Type 1; Orphan Drug Designation to BDC-1001 for Gastric Cancers
AbbVie Presents Results from Phase III CANOVA Study of Venetoclax in Patients with Relapsed or Refractory Multiple Myeloma AbbVie has released findings from its Phase III CANOVA trial, which assessed the safety and effectiveness of venetoclax (marketed as VENCLEXTA®/VENCLYXTO®) in combination with dexamethasone ...
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Sep 12, 2023
BMS’s LPA1 Antagonist; Alnylam’s KARDIA-1 Phase 2 Study; Day One Biopharma Sought FDA Approval for Tovorafenib; EMA Orphan Drug Designation to MaaT Pharma’s MaaT033; Lundbeck and Otsuka Announce Topline Results from Two Phase III Trials of Brexpiprazole + Sertraline; Phase III CheckMate – 227 Trial Show Durable, Long-Term Survival with Opdivo Plus Yervoy
Bristol Myers Squibb’s Investigational LPA1 Antagonist Reduces Rate of Lung Function Decline in Progressive Pulmonary Fibrosis Cohort of Phase II Study BMS-986278, a potential first-in-class oral lysophosphatidic acid receptor 1 (LPA1) antagonist, was studied in patients with progressive pulmonary fibrosis (PPF)...
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Sep 05, 2023
Daiichi Sankyo’s Trastuzumab Deruxtecan; ODD to Bexmarilimab for AML; Roche’ Alecensa; Ergomed Aims To Go Private; Tagraxofusp Receives ODD in Japan for BPCDN; FDA Fast Track Designation to Abliva’s KL1333
FDA Grants Breakthrough Therapy Designations to Trastuzumab Deruxtecan for HER2+ Solid Tumors, Including mCRC ENHERTU® (fam-trastuzumab deruxtecan-nxki) has been granted two additional Breakthrough Therapy Designations (BTDs) in the United States for the treatment of adult patients with unresectable or metastati...
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Aug 29, 2023
FDA Approves BMS’s Reblozyl for MDS; FDA Awards Orphan Drug Designation to NXC-201; Janssen Submits Supplemental NDA for Full Approval of BALVERSA; FDA Grants Fast Track Status to ALE.C04; FDA Orphan Drug Designation to Faron’s Bexmarilimab; FDA Clears IND Application for AHB-137
FDA Approves Bristol Myers Squibb’s Reblozyl as First-Line Treatment of Anemia in Adults with Lower-Risk MDS Who May Require Transfusions Bristol Myers Squibb announced that the Food and Drug Administration (FDA) has approved Reblozyl® (luspatercept-aamt) for the treatment of anemia in adult patients with very l...
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