News about "USFDA"

FDA Accepts Ionis' Zilganersen NDA for Priority Review in AxD

Ionis’ zilganersen NDA for Alexander disease has been accepted by the FDA for Priority Review, with a PDUFA target action date set for 22nd September, 2026, marking a key regulatory milestone for the potential treatment.

USFDA | 24/03/2026 | By News Bureau

IntraBio Submits sNDA for Levacetylleucine in Ataxia-Telangiectasia

IntraBio has submitted an sNDA to the US Food and Drug Administration (FDA) for Levacetylleucine, marking the first regulatory filing seeking approval for the treatment of Ataxia-Telangiectasia.

USFDA | 21/03/2026 | By News Bureau

Argo Biopharma's siRNA Therapy BW-20805 Gets FDA Fast Track for HAE

Argo Biopharma has received FDA Fast Track designation for BW-20805, with an ongoing global phase-II study in adult HAE patients and primary completion expected in the second half of 2026.

USFDA | 17/03/2026 | By News Bureau

Zydus Gets USFDA Tentative Nod for Dapagliflozin Tablets

Zydus has received tentative approval from the US Food and Drug Administration (USFDA) for dapagliflozin tablets in 5 mg and 10 mg strengths, paving the way for the company’s entry into the US market subject to final approval.

USFDA | 06/02/2026 | By News Bureau |  102

US FDA Grants Orphan Drug Designation to IFx-2.0 of TuHURA Biosciences

TuHURA Biosciences has received FDA Orphan Drug Designation for IFx-2.0 to treat stage IIB–IV cutaneous melanoma, based on data from its completed Phase I study published in Molecular Therapeutics.

USFDA | 04/02/2026 | By News Bureau |  113

Pharming Gets Complete Response Letter from US FDA for Joenja

The US FDA issued a Complete Response Letter to Pharming Group for the supplemental NDA for Joenja, citing the need for additional information to support its use in children aged four to 11 years with activated phosphoinositide 3-kinase delta syndrome, a rare primary immunodeficiency.

USFDA | 02/02/2026 | By News Bureau |  154

Life Biosciences Wins FDA IND Clearance for ER-100

The IND clearance allows Life Bio to initiate a clinical programme evaluating ER-100’s safety and potential to improve vision in patients with optic neuropathies.

USFDA | 29/01/2026 | By News Bureau |  1681

Immix Biopharma Gets US FDA Breakthrough Therapy Designation for NXC-201

Immix Biopharma has received US FDA Breakthrough Therapy Designation for its CAR-T candidate NXC-201, based on positive interim phase-II results from the NEXICART-2 study presented at the ASH 2025 annual meeting, with final data expected later this year followed by a planned Biologics Licence Application (BLA) submission.

USFDA | 29/01/2026 | By News Bureau |  119

FDA Grants Orphan Drug Status to BlueRock's OpCT-001

The ODD supports the clinical development of OpCT-001, the first iPSC-derived investigational cell therapy to enter human trials for Retinitis Pigmentosa (RP), a common inherited retinal disease marked by progressive loss of photoreceptor cells.

USFDA | 23/01/2026 | By News Bureau |  161

ImmunityBio Advances FDA Talks on ANKTIVA Resubmission

ImmunityBio said the FDA has requested additional information to potentially support a resubmission of the sBLA for ANKTIVA in BCG-unresponsive papillary bladder cancer, without requiring new clinical trials, with the company planning to submit the data within 30 days.

USFDA | 21/01/2026 | By News Bureau |  207