New Drug Designations

Shots:

PharmaShots' designation report provides a concise overview of several drugs and their designations by the FDA, NMPA and EMA. August month’s report includes designations allotted to 10 small molecules, 9 biologics, 7 cell & gene therapies, 1 vaccine, 2 radiopharmaceutical, 1 peptide, 1 antineoplastic and 2 devices
In August 2024, Invenra’s INV724 received the US FDA’s ODD & RPDD for neuroblastoma and ReAlta’s RLS-0071 was designated with the US FDA’s ODD & FTD for Steroid-Refractory Acute Graft-versus-Host Disease
We have compiled a list of a total of 30 drugs and 2 devices, awarded with designations by multiple regulatory bodies in August 2024

Opaganib

The designation has been granted to opaganib for treating neuroblastoma
Opaganib is a first-in-class, SPHK2 inhibitor having anticancer, anti-inflammatory & antiviral properties. It targets diseases such as prostate cancer, cholangiocarcinoma, obesity-related syndromes, gastrointestinal acute radiation syndrome (GI-ARS), sulfur mustard exposure and viruses like COVID-19 & Ebola

Alemtuzumab (CLLS52)

Alemtuzumab is being assessed under the P-I/IIa (BALLI-01) dose-escalation & expansion trial for its safety, expansion, persistence and clinical activity to treat r/r B- cell ALL

RLS-0071

The US FDA has granted Orphan Drug Designation (ODD) & Fast Track Designation (FTD) to the company’s RLS-0071 for treating steroid-refractory acute graft-versus-host disease (aGvHD) in hospitalized individuals
RLS-0071 is presently an ongoing P-II open-label study for treating hospitalized individuals who have steroid-refractory aGvHD
Additionally, the company is presently carrying out a P-II study for RLS-0071 in newborns suffering from hypoxic-ischemic encephalopathy (HIE) and in hospitalized individuals experiencing acute exacerbations of chronic obstructive pulmonary disease

INV724

The US FDA has granted ODD and also a RPDD to INV724 for neuroblastoma
INV724 aims to reduce severe pain from GD2-targeted therapies and has shown strong therapeutic potential and developability in preclinical studies
INV724 is being developed using Invenra's B-Body Bispecific Platform in partnership with the University of Wisconsin. It targets GD2 & B7-H3 tumor antigens with high specificity for neuroblastoma

Rivoceranib + Camrelizumab

The combination of rivoceranib & camrelizumab has been granted ODD as a 1L treatment of unresectable hepatocellular carcinoma (uHCC)
Rivoceranib is a small-molecule TKI that inhibits VEGFR, a key driver of tumor angiogenesis
Camrelizumab (SHR-1210) is a humanized mAb that targets PD-1 receptor, blocking the PD-1/PD-L1 pathway to treat various solid and hematological cancers

SLS009

The ODD has been granted to SLS009 for treating r/r peripheral T-cell lymphomas (PTCL)
In the P-I study for r/r hematological malignancies, SLS009 showed favorable safety, with a 36.4% response rate in PTCL patients and1 patient remained in treatment for over 56wks.

Soquelitinib

The US FDA has granted FTD to soquelitinib for treating r/r peripheral T cell lymphoma (PTCL) in patients treated with at least 2L of systemic therapy
Soquelitinib is a small molecule that works by inhibiting interleukin-2-inducible T cell kinase enzyme found in T cells, leading to T cell & NK cell immune function

TNX-102

Tonix Pharmaceuticals’ TNX-102 (cyclobenzaprine HCl sublingual tablets) has received the US FDA’s FTD to manage fibromyalgia
Its P-III study demonstrated improved fibromyalgia pain & sleep quality, low fatigue plus improved overall fibromyalgia symptoms & function. The drug was well-tolerated with most common AEs being transient sensations associated with dissolving of tablets
Tonix will submit NDA to the US FDA during H2’24 and will request for priority review to expedite application

AR882

The US FDA has granted FTD to AR882 (next gen. URAT1 inhibitor) for treating clinically visible tophi in patients with gout. Its P-III study is ongoing

[18F] PI-2620

The US FDA has granted fast track designation to [18F] PI-2620 injection for imaging in neurodegenerative disorders such as Alzheimer’s disease (AD), progressive supranuclear palsy (PSP), and corticobasal degeneration (CBD)
[18F] PI-2620 is being assessed under the P-III clinical evaluation for detecting tau pathology in Alzheimer’s disease, with ongoing research in other neurodegenerative diseases by academic researchers & other drug development studies
[18F] PI-2620 is a PET imaging agent developed under a partnership b/w AC Immune and LMI, with LMI securing exclusive rights for research, development & commercialization of Tau PET tracers generated within the discovery program

RLS-0071

The US FDA has granted Orphan Drug Designation (ODD) & Fast Track Designation (FTD) to the company’s RLS-0071 for treating steroid-refractory acute graft-versus-host disease (aGvHD) in hospitalized individuals
RLS-0071 is presently an ongoing P-II open-label study for treating hospitalized individuals who have steroid-refractory aGvHD
Additionally, the company is presently carrying out a P-II study for RLS-0071 in newborns suffering from hypoxic-ischemic encephalopathy (HIE) and in hospitalized individuals experiencing acute exacerbations of chronic obstructive pulmonary disease

BGB-16673

The US FDA has granted FTD designation based on the data from P-I/II study, highlighted at EHA 2024, its tolerability and favorable efficacy in heavily pretreated patients with r/r CLL/SLL
BGB-16673 is a BTK targeting chimeric degradation activation compound (CDAC) developed to degrade BTK (wild type and mutant) in patients with progressive disease

Deltacel (KB-GDT-01)

The US FDA has granted FTD to Deltacel (gamma delta T-cell therapy) in addition to low-dose radiation therapy for treating metastatic NSCLC patients whose disease progressed after 2L of SoC, based on the data from P-I (Deltacel-01) study showing favorable safety & efficacy
The P-I (Deltacel-01) trial aims to assess the safety & tolerability of Deltacel + low dose radiation for treating stage 4 NSCLC patients, with 1EP as safety & 2EPs as ORR, PFS, OS, time to progression, TTR & DCR

AC699

The US FDA has granted FTD to AC699 for treating ER+/HER2-, ESR1-mutated advanced or metastatic breast cancer patients whose disease progressed on or after at least 1L of endocrine-based therapy
AC699 is being assessed under the P-I trial for its safety, tolerability, PK & preliminary anti-tumor activity to treat ER+/HER2- locally advanced or metastatic breast cancer. Study showed an ORR of 50%, as highlighted at ASCO 2024
AC699 is a chimeric degrader of ERα. It has shown potent and selective degradation of both wildtype and mutant ERα, along with anti-tumor activity in ER-positive animal models

ABA-101

Based on an in vivo preclinical trial, the US FDA has granted fast-track designation to the company’s ABA-101 to treat progressive MS
In trial, the drug was well tolerated and demonstrated antigen-dependent Treg functionality, increased anti-inflammatory cytokine production, reduced inflammatory cytokine production, and therapeutic effects
ABA-101 is an autologous Treg therapy using the patient’s own Tregs to express a T cell receptor (TCR) that specifically targets immunogenic myelin fragments in the CNS, providing a favorable safety profile and localized anti-inflammatory effects at the disease site. Recently, the drug received IND approval, and the company plans to initiate its P- I trial

Imfinzi

The US FDA has accepted and granted priority review to the sBLA of Imfinzi for treating LS-SCLC patients with disease progression after Pt-based cCRT. The PDUFA date is expected in Q4’24. It also received BTD for the same indication
The submission was based on the P-III (ADRIATIC) study assessing Imfinzi alone and Imfinzi + Imjudo vs PBO in LS-SCLC patients (n=730) who had not progressed after cCRT. Results were highlighted at ASCO 2024
Study showed reduced death risk by 27%, mOS of 55.9mos. vs 33.4mos. with 57% vs 48% remaining alive at 3yrs., reduced disease progression or death by 24% & mPFS of 16.6mos. vs 9.2mos., with 46% vs 34% patients not experiencing disease progression at 2yrs.

Enhertu

The US FDA has granted BTD to Enhertu (fam-trastuzumab deruxtecan-nxki) for unresectable or metastatic HER2 low or HER2 ultralow breast cancer who have treated with at least 2L endocrine therapy in the metastatic indication or 1L of endocrine therapy if they experienced disease progression within 6mos.
The designation was based on the P-III (DESTINY-Breast06) study assessing Enhertu (5.4 mg/kg) vs investigator’s choice of CT (N=866, HER2 low: n=713 for and HER2 ultralow: n=153); significant improvement in 1EP: PFS. 2EPs: PFS by BICR, OS in overall population, OS in HER2 low population, and other 2Eps: ORR, DoR, etc.
This data was presented at ASCO 2024. Enhertu is jointly developed and commercialized by Daiichi Sankyo and AstraZeneca

124I-Evuzamitide (AT-01)

The US FDA has granted BTD to the company’s non-invasive pan-amyloid PET imaging agent, 124I-evuzamitide (AT-01), intended for the diagnosis of cardiac amyloidosis. It has also been designated with the US FDA & EC’s ODD for ATTR & AL amyloidosis
The designation was based on the results from Attralus-sponsored & investigator-initiated trials assessing 124I-evuzamitide among cardiac amyloidosis patients. Its P-III trial is expected during H1’25
124I-evuzamitide, labeled with iodine-124, is an amyloid-specific imaging agent intended to detect all kinds of systemic amyloidosis. It can identify amyloid deposits such as ATTR & AL, in organs like the heart, kidney, liver, and spleen as demonstrated in clinical evaluations

GSK5764227

The US FDA has granted BTD to GSK’227, a B7-H3-targeted ADC, for ES-SCLC with disease progression on or after Pt-based CT, and GSK holds exclusive development and commercialization rights worldwide (excluding China) from Hansoh Pharma
The BTD was based on early clinical evidence from the P-I (ARTEMIS-001) study assessing safety, tolerability & preliminary anti-tumor activity in r/r ES-SCLC individuals (n>200), conducted by Hansoh Pharma
GSK’227 aka. HS-20093 is now being developed by Hansoh Pharma. An ongoing P-I/II study for various solid tumors in China. Additionally, GSK plans to initiate a P-I/II global study in H2’24 to support a registrational pathway for GSK’227

9MW2821

The NMPA has granted BTD to 9MW2821 (nectin-4 targeting ADC) for treating locally advanced or metastatic urothelial carcinoma in patients who failed on Pt-based CT and PD-(L)1 inhibitor therapy

CHIKV VLP Vaccine

The US FDA has accepted & granted priority review to the BLA of CHIKV VLP vaccine in patients (≥12yrs.) for prevention of chikungunya virus infection, with the decision anticipated on Feb 14, 2025
Following the CHMP’s positive opinion in Feb 2024, the EMA is reviewing the MAA of the vaccine under accelerated assessment, seeking EC’s approval in H1’25
CHIKV VLP, an adjuvanted VLP-based vaccine, is a single-dose vaccine intended for active immunization to prevent CHIKV infection. It will be manufactured as a pre-filled syringe, if approved

Imfinzi

The US FDA has accepted and granted priority review to the sBLA of Imfinzi for treating LS-SCLC patients with disease progression after Pt-based cCRT. The PDUFA date is expected in Q4’24
The submission was based on the P-III (ADRIATIC) study assessing Imfinzi alone and Imfinzi + Imjudo vs PBO in LS-SCLC patients (n=730) who had not progressed after cCRT. Results were highlighted at ASCO 2024
Study showed reduced death risk by 27%, mOS of 55.9mos. vs 33.4mos. with 57% vs 48% remaining alive at 3yrs., reduced disease progression or death by 24% & mPFS of 16.6mos. vs 9.2mos., with 46% vs 34% patients not experiencing disease progression at 2yrs.

Diazoxide Choline (DCCR)

The US FDA has accepted and granted priority review (PDUFA: Dec 27, 2024) to the NDA of DCCR for treating Prader-Willi syndrome (PWS) patients (≥4yrs.) having hyperphagia

Mirdametinib

The US FDA has accepted and granted priority review (PDUFA: Feb 28, 2025) to the NDA of mirdametinib for treating adult and pediatric patients with NF1-PN, with EMA validating its MAA for the same
The submissions were supported by P-IIb (ReNeu) study assessing the efficacy, safety & tolerability of mirdametinib (2mg/m^2, BID) in patients (n=114, ≥2yrs.) with inoperable NF1-associated PN
Results, highlighted at ASCO 2024, showed strong ORR, durable responses, improved pain & quality of life plus a manageable safety profile in both adult and pediatric arms

Ivonescimab

The NMPA has granted priority review to the sNDA of ivonescimab (PD-1/VEGF bispecific antibody) as monotx. for 1L treatment of PD-L1+ve advanced or metastatic NSCLC
The submission was based on HARMONi-2 trial, showing improved PFS when compared to Keytruda
Akeso is conducting 5 P-III studies of ivonescimab, with 2 global MRCTs and 4 registrational trials against anti-PD-1 therapies. The drug is also being assessed for 16 indications, incl. gastrointestinal, liver & colorectal cancers

ATSN-201

The US FDA has granted RPDD to ATSN-201 for treating X-linked retinoschisis (XLRS). The company will receive a priority voucher on approval that could be sold or used for developing other internal product
ATSN-201 is currently being assessed under the dose escalation & expansion P-I/II (LIGHTHOUSE) study for treating XLRS caused due to RS1 gene mutations in male patients (≥6yrs.). Recruitment is underway
ATSN-201, a gene therapy, uses AAV.SPR (novel spreading capsid) for therapeutic gene expression in central retinal photoreceptors without the risks of foveal detachment. Preclinical studies in non-human primates showed that AAV.SPR induces transgene expression beyond subretinal injection areas

OPGx-LCA5

OPGx-LCA5 is being investigated under P-I/II study at the University of Pennsylvania for its safety & preliminary efficacy in patients (n=15) with inherited retinal degeneration caused by biallelic LCA5 mutations
OPGx-LCA5 is an AAV8 vector developed to transfer a functional LCA5 gene to the outer retina

META-001-PH

The US FDA has granted RPDD to the company’s META-001-PH (oral, daily) for treating primary hyperoxaluria (PH)
META-001-PH has reduced urinary oxalate excretion by up to 80% and depicted favorable safety & tolerability in non-clinical studies and is currently under IND-enabling toxicology studies. Its P-I among healthy individuals is anticipated during H1’25 in Australia
META-001-PH is a small molecule that has been developed under partnership using META's chemistry team and XtalPi's AI drug discovery team, from initial screening to nomination

SAT-3247

The US FDA has granted RPDD to SAT-3247 for treating DMD. Its P-I study is expected to begin in Q3’24
SAT-3247 is a first-in-class small molecule developed to re-establish muscle regeneration and repair, independent of dystrophin or exon mutation status

INV724

INV724 has also received ODD for neuroblastoma
INV724 aims to reduce severe pain from GD2-targeted therapies and has shown strong therapeutic potential and developability in preclinical studies
INV724 is being developed using Invenra's B-Body Bispecific Platform in partnership with the University of Wisconsin. It targets GD2 & B7-H3 tumor antigens with high specificity for neuroblastoma

CERTA Access System

The US FDA has granted BDD designation to the CERTA Access System for central venous catheterization (CVC)
The system consists of robotics and imaging to enhance the accuracy, safety & consistency of accessing targeted anatomy. The company is on track for its approval

eShunt System

The US FDA has granted BDD to eShunt System for treating normal pressure hydrocephalus (NPH), based on results from the pivotal study
CereVasc received an IDE to conduct the system’s STRIDE pivotal trial for NPH. The recruitment expected to start during H2’24

Ixoberogene Soroparvovec (Ixo-vec)

The US FDA has granted RMAT designation to Ixo-vec for treating wet-AMD. Further updates will be provided in Q4’24
Ixo-vec is currently being assessed under P-II (LUNA) study and P-I (OPTIC) extension trial to treat neovascular or wet AMD

NGN-401

NGN-401 (AAV9 gene therapy) has received the US FDA’s RMAT designation for treating Rett syndrome, based on the preliminary results from ongoing P-I/II study. It was also selected for the US FDA’s START program
The interim efficacy results of low-dose arm are anticipated in Q4’25 and high-dose arm in H2’25

KYV-101

KYV-101 (autologous, fully human CD19 CAR T-cell product) has received the US FDA’s RMAT designation for treating progressive myasthenia gravis
KYV-101 is being assessed under P-I/II & P-II studies across the US & Germany for autoimmune diseases in rheumatology & neurology, as well as in investigator-conducted studies for various indications globally

References