Shots:
PharmaShots’ designation report provides a concise overview of several drugs and their designations by the FDA, MHRA and EC. This month’s report includes designations allotted to 7 small molecules, 4 biologics, 4 cell & gene therapy, 2 devices
Trellis Bioscience’s TRL1068 has received FTD and QIDP designations from the US FDA for the treatment of Infectious diseases
PharmaShots has compiled a list of a total of 33 drugs and 2 devices awarded with designations by multiple regulatory bodies in Feb 2024
Avutometinib
The ODD has been granted to avutometinib alone or in addition with defactinib to treat recurrent low-grade serous ovarian cancer (LGSOC)
The drug is currently under P-III (RAMP 301) clinical evaluation with defactinib vs standard CT or hormonal therapy as a treatment of recurrent LGSOC
The P-II (RAMP 201) registration-directed study also investigates avutometinib + defactinib for recurrent LGSOC. The patient recruitment in the dose optimization, expansion phase & low-dose evaluation arms has been completed
The company anticipates the rolling NDA submission for accelerated approval in H1’24 and its launching in 2025
CABA-201
Sponsor
Cabaletta Bio
Indication
Systemic Sclerosis
Phase
P-I/II
MOA
T lymphocyte replacements
RoA
IV
Approval Authority
FDA
Date
Mar 20, 2024
CABA-201 is being evaluated under the RESET (REstoring SElf-Tolerance) program consisting of 4 P-I/II studies for treating various autoimmune disorders incl. P-I/II (RESET-SSc) study
The P-I/II (RESET-SSc) program comprises a total of 9 arms with all arms starting at a dose of 1×10^6 cells/kg without the need of dose escalation
Cabaletta has gained the US FDA’s IND clearance of CABA-201 across various autoimmune conditions like systemic lupus erythematosus (SLE), myositis, systemic sclerosis (SSc), and generalized myasthenia gravis (gMG)
CABA-201 aims to temporarily deplete CD19+ B cells with a single infusion, leading to immune system reset for a lasting remission off therapy for autoimmune disease patients
Source: https://www.cabalettabio.com/news-media/press-releases/detail/107/cabaletta-bio-announces-fda-granted-orphan-drug-designation
Felzartamab – Biologic (Priyanka)
Sponsor
HI-Bio
Indication
Antibody-mediated rejection (AMR) in Kidney Transplantation
Phase
P-II
MOA
Antibody-dependent cell cytotoxicity
ROA
IV
Approval Authority
FDA
Date
Mar 21, 2024
HI-Bio’s Felzartamab has received ODD from the US FDA for AMR treatment in kidney transplant recipient and was already designated with the US FDA’s BTD for primary membranous nephropathy
In 2022, the company in-licensed felzartamab from MorphoSys and possess exclusive global rights excl. Greater China
Felzartamab is a monoclonal antibody that targets CD38 to improve clinical outcomes in a wide range of pathogenic antibody mediated disorders. It is being explored for other severe immune-mediated diseases, incl. IgA nephropathy (IgAN), lupus nephritis (LN) & primary membranous nephropathy (PMN)
Source: HI-Bio (hibio.com)
Utidelone Injectable (UTD1) – Small Molecule
Sponsor
Biostar Pharma
Indication
Breast Cancer Brain Metastasis
Phase
P-II
MOA
Mitosis inhibitors
RoA
IV
Approval Authority
FDA
Date
Mar 29, 2024
The ODD was supported by the two P-II studies, one of them (n=17) assessing utidelone injectable + etoposide & bevacizumab resulted in 73% CNS ORR & 91% CNS CBR while the other one (n=46) evaluated utidelone injectable + bevacizumab & showed mPFS of 7.7mos. & 12mos. OS rate of 74.4% in HER2 -ve BCBM patients
The company further anticipates the expansion of utidelone’s clinical evaluations for treating lung cancer brain metastasis & glioma based on its potential to cross BBB due to insusceptibility to P-glycoprotein-mediated efflux & physicochemical features
Source: https://www.prnewswire.com/news-releases/73-cns-orr-fda-granted-odd-to-utidelone-injectable-utd1-from-biostar-pharma-for-the-treatment-of-breast-cancer-brain-metastasis-302103497.html
A2B530 – Cell & Gene Therapy – Oncology
Sponsor
A2 Biotherapeutics
Indication
Colorectal Cancer
Phase
P-I
MOA
T lymphocyte replacements
RoA
IV
Approval Authority
FDA
Date
Mar 04, 2024
The designation has been granted to A2B530 for treating germline heterozygous HLA-A*02(+) patients with colorectal cancer having carcinoembryonic antigen (CEA) expression and loss of HLA-A*02 expression
The company is evaluating A2B530 in the P-I/II (EVEREST-1) trial for its safety and efficacy to treat colorectal, pancreatic and non-small cell lung cancers
A2B530, based on Tmod platform, is an autologous logic-gated cell therapy which comprises of an activator targeting CEA and a blocker targeting HLA-A*02
Source: https://www.a2bio.com/a2-bio-receives-fda-orphan-drug-designation-for-novel-cell-therapy-program-a2b530-in-colorectal-cancer/
UNI-494 – Small Molecule (Priyanka)
Sponsor
Unicycive Therapeutics
Indication
Acute Kidney Injury
Phase
P-I
MOA
ATP-sensitive mitochondrial potassium channel activator
RoA
IV
Approval Authority
FDA
Date
Mar 04, 2024
The US FDA granted ODD to prevent delayed graft function (DGF) in kidney transplant patients
The company is currently assessing the drug under the P-I dose-ranging trial for its safety among healthy volunteers in UK and is expected to be concluded in H2’24
The preclinical data & P-I design were highlighted at the 29th International Conference on Advances in Critical Care Nephrology AKI and CRRT 2024
UNI-494, a cryoprotective agent, activates KATP channels in mitochondria restoring its function and prevent damage caused by ischemia/reperfusion injury. It is patent protected across the US & the EU
Source: https://ir.unicycive.com/news/detail/62/unicycive-therapeutics-announces-orphan-drug-designation
Cambritaxestat (IOA-289)- Small Molecules (Priyanka)
Sponsor
iOnctura
Indication
Pancreatic Cancer
Phase
P-I
MOA
Autotaxin inhibitor
ROA
Oral
Approval Authority
FDA
Date
Mar 07, 2024
The company is assessing cambritaxestat under the P-I (AION-02) trial in combination with nab-paclitaxel and gemcitabine for the treatment of metastatic pancreatic cancer
Source: odd-for-cambritaxestat-final.pdf (ionctura.com)
TERN-701 – Small Molecule
Sponsor
Terns Pharmaceuticals
Indication
Chronic Myeloid Leukemia
Phase
P-I
MOA
BCR-ABL Tyrosine Kinase Inhibitor
RoA
Oral
Approval Authority
FDA
Date
Mar 11, 2024
The company’s TERN-701 is under P-I clinical evaluation for treating CML
Interim results from initial dose escalation arms are anticipated in H2’24
TERN-701 is an in-house allosteric BCR-ABL tyrosine kinase inhibitor (TKI) which is presented being assessed for the treatment of CML
Source: https://ir.ternspharma.com/news-releases/news-release-details/terns-pharmaceuticals-announces-orphan-drug-designation-granted
P-BCMA-ALLO1 – Cell & Gene Therapy
Sponsor
Poseida Therapeutics
Indication
R/R Multiple Myeloma
Phase
P-I
MOA
T lymphocyte replacements
RoA
IV
Approval Authority
FDA
Date
Mar 13, 2024
Developed in partnership with Roche, P-BCMA-ALLO1 is being assessed under P-I, the results of which were highlighted at ASH 2023
The data demonstrated a well-tolerated and favorable safety profile in the ITT population without the need for bridging CT. Preliminary data showed the conversion of TSCM-rich CAR-T cells into cell-killing effector T cells that were sustained for at least 6wks. post treatment
The new data from recently enrolled patients were highlighted at AACR 2024 and the additional data is anticipated in H2’24
Source: https://investors.poseida.com/news-releases/news-release-details/poseida-therapeutics-announces-fda-orphan-drug-designation
LYT-200 – Biologic (Priyanka)
Sponsor
PureTech Health
Indication
Acute myeloid leukemia (AML)
Phase
P-Ib
MOA
Galectin inhibitor
ROA
IV
Approval Authority
FDA
Date
Mar 13, 2024
The company is assessing LYT-200 under P-Ib trial as monotx. And in combination with venetoclax and hypomethylating agents (HMA) for treating both AML & MDS. The initial data was revealed in 2023 and updated results will be highlighted at future conferences in 2024
LYT-200, a human IgG4 mAb, targets galectin-9, depicted direct cytotoxic, anti-leukemic effects & synergized with SoC CT & venetoclax in its preclinical studies
Source: PureTech Receives Orphan Drug Designation for LYT-200 in Acute Myeloid Leukemia (puretechhealth.com)
AP303 – Small Molecule
Sponsor
Alebund Pharmaceuticals
Indication
Autosomal Dominant Polycystic Kidney Disease
Phase
P-I
MOA
Undisclosed
RoA
Oral
Approval Authority
FDA
Date
Mar 29, 2024
The company’s AP303 has shown renal survival improvement in an ADPKD mouse model.
Following the completion of FIH study among healthy subjects in Australia, it will now be evaluated under the P-II study for ADPKD
Source: http://www.alebund.com/release/ap303-receives-fda-odd-for-treatment-of-adpkd/
ImmCelz (CELZ-101) – Cell therapy (Priyanka)
Sponsor
Creative Medical Technology Holdings
Indication
Type 1 Diabetes
Phase
Preclinical
MOA
Cell replacements
ROA
Undisclosed
Approval Authority
FDA
Date
Mar 06, 2024
CELZ-101 received ODD from the US FDA to prevent allograft rejection in patients undergoing pancreatic islet cell transplantation, hope for type 1 diabetes individuals
CELZ-101 is a cell-based immunotherapy that uses the patient’s own Tregs to suppress rejection and potentially eliminate the need for lifelong immunosuppression after transplant
Source: https://www.businesswire.com/news/home/20240306397066/en/Creative-Medical-Technology-Holdings-Inc.-Receives-FDA-Orphan-Drug-Designation-for-CELZ-101-in-Key-Transplantation-Therapy
Tinengotinib- Small Molecule (Priyanka)
Sponsor
TransThera
Indication
Biliary Tract Cancer
Phase
P-III
MOA
FGFR inhibitor
ROA
Oral
Approval Authority
EMA
Date
Mar 08, 2024
TransThera’s, tinengotinib has received ODD from the EMA for the treatment of biliary tract cancer (BTC) and was already designated with the US FDA’s ODD and FTD for CCA and in Jul 2023, it received the NMPA’s BTD
The company will now assess tinengotinib under the P-III (FIRST-308) trial for its safety & efficacy among cholangiocarcinoma (CCA) patients with FGFR-alteration, pretreated with CT- & r/r to FGFR inhibitor. The first patient has been dosed across the US in Dec 2023
Additionally, the P-/II clinical evaluation depicted the drug’s clinical benefit among the same patient population. The results were highlighted at ESMO 2023 & ASCO GI 2024
Source: https://www.prnewswire.com/news-releases/transthera-announced-global-phase-3-clinical-trial-for-cholangiocarcinoma-authorized-in-the-european-union-and-orphan-drug-designation-for-tinengotinib-to-treat-biliary-tract-cancer-granted-by-european-medicines-agency-302084086.html
Nipocalimab – Biologic (Priyanka)
Sponsor
Johnson & Johnson
Indication
Fetal Neonatal Alloimmune Thrombocytopenia (FNAIT)
Phase
P-III
MOA
Neonatal Fc receptor antagonists
ROA
IV
Approval Authority
FDA
Date
Mar 26, 2024
J&J’s nipocalimab has received the US FDA’s FTD to reduce the risk of fetal neonatal alloimmune thrombocytopenia (FNAIT) in pregnant women. It had also received the US FDA’s ODD for FNAIT in Dec 2023
The P-III (FREESIA) study assessing nipocalimab for treating FNAIT is underway
Nipocalimab is being studied hemolytic disease of the fetus and newborn (HDFN) under P-III studies
Source: Johnson & Johnson’s nipocalimab granted U.S. FDA Fast Track designation to reduce the risk of fetal neonatal alloimmune thrombocytopenia (FNAIT) in alloimmunized pregnant adults (jnj.com)
EP0031/A400 (Priyanshu Mehta)
Sponsor
Ellipses Pharma
Indication
NSCLC
Phase
P-I/II
MOA
RET inhibitor
RoA
Oral
Approval Authority
FDA
Date
Mar 05, 2024
EP0031/A400 received FTD for the treatment of RET-fusion positive NSCLC, it also received ODD in Nov 2023
Data from interventional P-I/II showed tumour responses in NSCLC patients who were previously treated with first-generation SRIs.
Developed jointly by Ellipses Pharma (NCT05443126) globally and Sichuan-Kelun Biotech (NCT05265091) in China
Source: https://ellipses.life/ellipses-next-generation-selective-ret-inhibitor-ep0031-a400-granted-fast-track-designation-by-fda/
LYT-200 – Biologic
Sponsor
PureTech Health
Indication
Head and Neck Cancers
Phase
P-I/II
MOA
Galectin inhibitor
ROA
IV
Approval Authority
FDA
Date
Mar 11, 2024
The US FDA has granted FTD to LYT-200, human IgG4 mAb targeting galectin-9, in addition with tislelizumab to treat recurrent/metastatic HNSCC
The company’s LYT-200 is under the P-I/II trial as monotx. & combined with tislelizumab for advanced/metastatic solid tumors incl. HNSCC in which it demonstrated disease control, anti-tumor activity & a favorable safety profile across all cohorts
LYT-200 is also being investigated in the P-Ib study as monotx. & in combination with venetoclax & hypomethylating agents for treating hematological malignancies such as AML & high-risk myelodysplastic syndrome in which it showed a favorable safety & tolerability profile as well as potential clinical activity
Source: https://news.puretechhealth.com/news-releases/news-release-details/puretech-receives-fda-fast-track-designation-lyt-200-head-and
Elritercept (KER-050) – Recombinant fusion proteins (Priyanshu)
Sponsor
Keros Therapeutics
Indication
Myelodysplastic Syndromes (MDS)
Phase
P-II
MOA
TGF-ß Inhibitor
RoA
SC
Approval Authority
FDA
Date
Mar 14, 2024
The FTD has been granted to elritercept for the treatment of anemia in low or moderate MDS patients
KER-050 is an engineered ligand trap, combining the activin receptor type IIA ligand-binding domain with the human antibody’s Fc domain
Source: Keros Therapeutics Announces U.S. FDA Fast Track Designation for KER-050 in Lower-Risk Myelodysplastic Syndromes – Keros Therapeutics (kerostx.com)
PT886 – Antineoplastics (Priyanka)
Sponsor
Phanes Therapeutics
Indication
Pancreatic adenocarcinoma
Phase
P-I
MOA
CD47 antigen inhibitors
ROA
IV
Approval Authority
FDA
Date
March 6, 2024
PT886 has also received the US FDA’s ODD for the treatment of pancreatic cancer in 2022
The P-I (TWINPEAK) study is currently assessing the safety, tolerability, PK/PD and preliminary efficacy of PT886 for treating locally advanced or metastatic G/GEJ and pancreatic cancers progressed post standard therapy or for which standard therapy was ineffective, intolerable or inappropriate
PT886 is a bispecific antibody, developed through PACbody and SPECpair platforms, targets claudin 18.2 and CD47 for treating G/GEJ and pancreatic adenocarcinomas
Source: Phanes Therapeutics’ PT886 granted Fast Track designation for the treatment of patients with metastatic claudin 18.2-positive pancreatic adenocarcinoma by the FDA – Phanes Therapeutics, Inc
MM120 – Small Molecule (Priyanka)
Sponsor
MindMed
Indication
Generalized Anxiety Disorder (GAD)
Phase
P-IIb
MOA
Serotonin 5-HT2A receptor agonists
ROA
Oral
Approval Authority
FDA
Date
Mar 07, 2024
The BTD was based on the P-IIb (MMED008) study for GAD patients (n=198) who received MM120 (25, 50, 100 or 200µg) vs PBO. The full analysis set (FAS) for the trial comprised 194 individuals, all with at least one valid post-baseline HAM-A score
At wk.12, MM120 showed a 7.7-point improvement vs PBO (-21.9 vs -14.2), clinical response rate was 65% & clinical remission rate was 48% that sustained to wk.12. CGI-S scores shifted from 4.8 to 2.2 by wk.12, indicating substantial improvement.
The company anticipates End-of-P-II meeting with the US FDA in H1’24 and commencement of P-III study in the H2’24
Source: https://ir.mindmed.co/news-events/press-releases/detail/137/mindmed-receives-fda-breakthrough-therapy-designation-and-announces-positive-12-week-durability-data-from-phase-2b-study-of-mm120-for-generalized-anxiety-disorder
CYB003 – Antidepressants
Sponsor
Cybin
Indication
Major Depressive Disorder
Phase
P-II
MOA
Serotonin 5-HT2A receptor agonists
RoA
Oral
Approval Authority
FDA
Date
Mar 13, 2024
The company’s CYB003 has received BTD as an adjunctive treatment of major depressive disorder supported by the data from CYB003’s (12mg or 16mg) P-II study in MDD
The results demonstrated a mean reduction of ~22 points in MADRS total score from baseline with both the doses, ~75% of patients responded after receiving 2 doses of 16mg and 60% (12mg) & 75% (16mg) of them were in remission from depression after 2 doses
Source: https://ir.cybin.com/investors/news/news-details/2024/Cybin-Receives-FDA-Breakthrough-Therapy-Designation-for-its-Novel-Psychedelic-Molecule-CYB003-and-Announces-Positive-Four-Month-Durability-Data-in-Major-Depressive-Disorder/default.aspx
PGN-EDO51 – Drug conjugates (Priyanshu)
Sponsor
PepGen
Indication
Duchenne Muscular Dystrophy (DMD)
Phase
P-II
MOA
Skip exon 51 of the dystrophin transcript
RoA
IV
Approval Authority
FDA
Date
Mar 13, 2024
PGN-EDO51 received ODD and RPDD for treatment of DMD patients with exon 51 skipping mutation
PepGen is currently evaluating safety and efficacy of PGN-ED051 in ongoing CONNECT1 P-II trial. PepGen is planning to initiate recruitment for the P-II CONNECT2 study later in 2024
PGN-EDO51 is designed to skip exon 51 of the dystrophin transcript, an established therapeutic target for approximately 13% of DMD patients, thereby aiming to restore the open reading frame and enabling the production of a truncated, yet functional dystrophin protein.
Source:https://www.globenewswire.com/en/news-release/2024/03/13/2845286/0/en/PepGen-Receives-U-S-FDA-Orphan-Drug-and-Rare-Pediatric-Disease-Designations-for-PGN-EDO51-for-the-Treatment-of-Duchenne-Muscular-Dystrophy.html
LSTA1- Antineoplastics (Priyanka)
Sponsor
Lisata Therapeutics
Indication
Osteosarcoma
Phase
P-IIa
MOA
Capillary permeability stimulants
ROA
IV
Approval Authority
FDA
Date
Mar 21, 2024
Lisata Therapeutics received RPDD from the US FDA for LSTA1 for the treatment of Osteosarcoma
LSTA1, has demonstrated favourable safety, tolerability, and activity in clinical study for pancreatic cancer. It works by improving delivery of existing anti-cancer drugs to tumors making them more effective
LST1A has already received ODD for pancreatic cancer in the US and for glioblastoma in EU
Source: https://ir.lisata.com/news-releases/news-release-details/lisata-therapeutics-announces-us-fda-rare-pediatric-disease
TNX-2900 – Analgesics (Priyanka)
Sponsor
Tonix Pharmaceuticals
Indication
Prader-Willi Syndrome
Phase
P-II
MOA
Oxytocin receptor agonists
ROA
Intranasal spray
Approval Authority
FDA
Date
Mar 25, 2024
Tonix Pharmaceuticals TNX-2900 has received RPDD from the US FDA for the treatment of Prader-Willi Syndrome
TNX-2900 targets PWS, a genetic disorder causing uncontrollable hunger and obesity in children
TNX-2900 was previously granted ODD by the FDA in 2022 for the treatment of PWS and IND application was approved by the FDA in 2023
Tonix Pharma may be eligible for transferable priority vouched if TNX-2900 is approved for marketing
Source: Tonix Pharmaceuticals Receives Rare Pediatric Disease Designation from the FDA for TNX-2900 for the Treatment of Prader-Willi Syndrome :: Tonix Pharmaceuticals Holding Corp. (TNXP)
TRL1068 – Biologic(Priyanka)
Sponsor
Trellis Bioscience
Indication
Infectious Diseases
Phase
P-I
MOA
Bacterial biofilm inhibitors
ROA
IV
Approval Authority
FDA
Date
March 6, 2024
Trellis Bioscience’s TRL1068 has Received FTD and QIDP designations from the US FDA for the treatment of Infectious diseases
TRL1068 targets prosthetic joint infections (PJIs) and other bacterial infections. It is designed to eliminate bacterial biofilms, making antibiotics more effective
P-I trial results shown TRL1068, is well tolerated with no serious side effects and eliminated bacterial biofilm in 7 days
Source: Trellis – Press Release (trellisbio.com)
P-Tau 217 (Priyanka)
Sponsor
Quanterix Corporation
Indication
Alzheimer’s Disease (AD)
Phase
N/A
MOA
N/A
ROA
N/A
Approval Authority
FDA
Date
Mar 04, 2024
Quanterix’s p-Tau 217 blood test has been granted BDD by the US FDA as an aid in diagnostic evaluation of Alzheimer’s Disease (AD)
P-Tau 217 offer a simpler and less invasive approach compared to traditional methods like PET scans or spinal taps
The test is not meant to be a standalone diagnostic tool, intended to be used alongside other diagnostic tools to improve the evaluation of Alzheimer’s risk, leading to earlier detection
Source: Quanterix Granted Breakthrough Device Designation from U.S. FDA for Blood-Based p-Tau 217 Test for Alzheimer’s Disease | Quanterix
Heart Disease Risk Staging System
Sponsor
Cleerly
Indication
Heart Attack Risk Assessment
Phase
N/A
MOA
N/A
RoA
N/A
Approval Authority
FDA
Date
Mar 05, 2024
Cleerly’s CAD staging system receives BDD from US FDA; This software device analyses actionable features of coronary atherosclerosis, stenosis and ischemia and aids physicians for assessment of patients at risk of MACE
It is a non-invasive imaging-based investigational software device that analyses important and actionable features of coronary atherosclerosis, stenosis and ischemia
The Cleerly CAD Staging System will be evaluated in the TRANSFORM study which will recruit asymptomatic heart disease patients with diabetes, pre-diabetes or metabolic syndrome
The Cleerly CAD Staging System was also accepted into the FDA’s Total Product Life Cycle Advisory Program (TAP) Pilot
Source: https://cleerlyhealth.com/press/breakthrough-device-designation-heart-disease-risk-staging-system
Neuroimmune modulation platform (Priyanka)
Sponsor
SetPoint Medical
Indication
Multiple sclerosis
Phase
N/A
MOA
N/A
ROA
Implant
Approval Authority
FDA
Date
Mar 13, 2024
SetPoint Medical received BDD from the US FDA for their investigational platform technology for RRMS followed by prior (2020) BDD designation for the treatment of RA
First-of-its-kind approach, this therapy targets pathways intended to promote remyelination, which is the regeneration of the protective layer around nerve Fibers damaged by Multiple sclerosis
This device is placed near vague nerve via small outpatient procedure on the left side of the neck and programmed. It later stimulates the vague nerve once daily to activate the inflammatory reflex for a systemic immune-restorative effect
This platform technology is also undergoing a pivotal clinical trial (RESET-RA) to investigate its effectiveness as a treatment for adults with moderate-to-severe rheumatoid arthritis
Source: SetPoint Medical Receives FDA Breakthrough Device Designation for its Neuroimmune Modulation Platform for the Treatment of Multiple Sclerosis
Almee (Priyanka)
Sponsor
Vicore Pharma
Indication
Pulmonary Fibrosis
Phase
N/A
MOA
N/A
ROA
Digital Therapy
Approval Authority
FDA
Date
Mar 19, 2024
Vicore Pharma’s Almee a digital therapy has received BDD from the US FDA for patients with Pulmonary Fibrosis
The COMPANION study demonstrated a significant 2.7-point improvement in GAD-7 scores (measuring anxiety) and a positive impact on quality of life, with a 4.4-point increase in KBILD scores for patients using Almee
It is software built for patients to be used on smartphones or tablets designed to increase beneficial actions and improve quality of life. This tool is based on CBT (Cognitive Behavior therapy) principle used as adjunct to treatment of anxiety
Source: Vicore Announces FDA Breakthrough Device Designation for AlmeeTM, a Digital Therapy for Patients with Pulmonary Fibrosis – Vicore Pharma
Freesolve BTK RMS (Priyanka)
Sponsor
Biotronik
Indication
Chronic limb-threatening ischemia (CLTI)
Phase
N/A
MOA
N/A
ROA
N/A
Approval Authority
FDA
Date
Mar 18, 2024
Biotronik’s Freesolve has received BDD from the US FDA for the treatment of CLTI
Freesolve BTK RMS leverages Biotronics’s experience with Freesolve RMS (Existing Technology) for coronary arteries, which uses a biocompatible magnesium alloy that degrades over time
Source: FDA Breakthrough Device Designation for BIOTRONIK Freesolve™ Below-the-Knee Resorbable Magnesium Scaffold (RMS) | Biotronik
DynamX BTK System (Priyanka)
Sponsor
Elixir Medical
Indication
Chronic limb-threatening ischemia (CLTI)
Phase
N/A
MOA
N/A
ROA
N/A
Approval Authority
FDA
Date
Mar 21, 2024
Elixir Medical’s DynamX BTK System received BDD from US FDA for the treatment of CLTI
The DynamX Bioadaptor has been shown to provide high acute lumen gain in coronary vessels and maintain such gain over time, which is a common challenge in existing below-the-knee (BTK) therapies
DynamX Bioadaptor is the first implant technology designed to support and then “uncage” a diseased vessel, promoting blood flow and preventing amputation
Source: elixirmedical.com/elixir-medical-receives-fda-breakthrough-device-designation-for-the-dynamx-btk-system-for-use-in-the-treatment-of-chronic-limb-threatening-ischemia-resulting-from-below-the-knee-arterial-disease/
Sebetralstat – Small Molecule (Priyanka)
Sponsor
KalVista Pharmaceuticals
Indication
Hereditary Angioedema (HAE)
Phase
P-III
MOA
Plasma kallikrein inhibitors
ROA
Oral
Approval Authority
MHRA
Date
March 12, 2024
KalVista Pharmaceuticals has received Promising Innovative Medicine (PIM) designation for sebetralstat from UK MHRA for the treatment of hereditary angioedema (HAE)
Sebetralstat has already received FTD and ODD from the U.S. FDA, as well as ODD and an approved Pediatric Investigational Plan from the European Medicines Agency (EMA)
Recent P-III data showcased clinically and statistically significant results, along with a good safety and tolerability profile
Source: KalVista Pharmaceuticals Awarded UK Promising Innovative Medicine (PIM) Designation for Sebetralstat | KalVista Pharmaceuticals
BJT-778 – Bioloic (Priyanka)
Sponsor
Bluejay Therapeutics
Indication
Chronic Hepatitis Delta Virus (HDV)
Phase
P-II
MOA
Hepatitis B surface antigen inhibitors
ROA
IV
Approval Authority
EMA
Date
March 25, 2024
Bluejay’s BJT-778 has received European Priority Medicine (PRIME) Designation from EMA for the treatment of Chronic Hepatitis Delta Virus
Bluejay’s application was supported by positive non-clinical and interim results from their ongoing P-I/II study
BJT-778 works as a high-potency antibody that neutralizes and helps clear HDV virus from the body
Source: Bluejay Announces Receipt of PRIME Designation from European Medicines Agency (EMA) for BJT-778 for the Treatment of Chronic Hepatitis Delta Virus Infection – Bluejay Therapeutics (bluejaytx.com)
PCRX-201 – Cell & Gene therapy (Priyanka)
Sponsor
Pacira BioSciences
Indication
Osteoarthritis
Phase
P-II
MOA
Hepatitis B surface antigen inhibitors
ROA
IV
Approval Authority
EMA
Date
March 13, 2024
Pacira BioSciences’s PCRX-201 has received RMAT designation from the US FDA for the treatment of Osteoarthritis and was also granted Advanced Therapy Medicinal Products (ATMP) designation by the European Medicines Agency in May 2023
PCRX-201 in P-I study showed (n=72) to be well tolerated with positive efficacy observed for at least 52 wks. 50% improved in the WOMAC pain and stiffness score and a meaningful improvement in KOOS functional assessment in PCRX-201+ steroid arm
The 52-week data have been accepted for presentation at OARSI 2024 and the company expects to present 104-week efficacy and safety data later in 2024
Source: Pacira BioSciences Announces PCRX-201 Granted Regenerative Medicine Advance Therapy (RMAT) Designation for the Treatment of Osteoarthritis of the Knee | Pacira BioSciences, Inc.
Related Post: New Drug Designations – February 2024
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LinkedIn Title: Get familiarized with the new designations being ascribed to the drugs now and then only at PharmaShots!
PharmaShots brings every month a Designation report, highlighting the designations allotted to drugs in various geographies.
This month, we have covered designations from the US, UK, and EU regions.
For a curated report on the designation of your need, reach out to us at connect@pharmashots.com
Stay Tuned for more updates!
