Drug Filing

Rocket Pharmaceuticals announces clearance of IND for RP-A501 gene therapy for Danon disease

Rocket Pharmaceuticals, Inc. (Nasdaq: RCKT) (“Rocket”), a leading U.S.-based multi-platform gene therapy company, today announces the clearance of the Company’s Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for RP-A501.

Lilly’s second immune-oncology candidate using Zymeworks’ Azymetric platform to start clinical trials

Zymeworks, a clinical-stage biopharmaceutical company developing multifunctional therapeutics, reported the achievement of a new development milestone in its collaboration with Eli Lilly and Company (“Lilly”).

Silence’s SLN124 gets orphan designation in Europe for β-Thalassemia

Silence Therapeutics’ lead candidate SLN124 has secured orphan drug designation in the European Union for the treatment of β-Thalassemia.

Pfizer’s tafamidis secures FDA priority review for ATTR-CM

Pfizer’s tafamidis meglumine form (20mg capsule) has secured priority review from the US Food and Drug Administration (FDA) for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM).

Ocular seeks FDA nod for expanded indication of Dextenza eye treatment

Ocular Therapeutix is seeking approval from the US Food and Drug Administration (FDA) for Dextenza (dexamethasone ophthalmic insert) to expand the current indication to include the treatment of ocular inflammation following ophthalmic surgery.

Momenta revises regulatory strategy for proposed Humira biosimilar

Momenta Pharmaceuticals has revised its regulatory strategy in the US for its anti-FcRn antibody M923, which is being proposed by the company as a biosimilar to Humira.

FDA fast tracks Novartis’ Zolgensma SMA gene replacement therapy

Novartis’ gene therapy Zolgensma (onasemnogene abeparvovec-xxxx), which was formerly known as AVXS-101, has been granted priority review by the US Food and Drug Administration (FDA) for the treatment of spinal muscular atrophy (SMA) Type 1.

AstraZeneca’s Fasenra secures FDA orphan drug designation for EGPA

AstraZeneca’s respiratory biologic Fasenra (benralizumab) has secured the orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for the treatment of Eosinophilic Granulomatosis with Polyangiitis (EGPA).

GSK seeks paediatric indication for Nucala in severe eosinophilic asthma

GlaxoSmithKline (GSK) has filed a supplemental Biologics License Application (sBLA) to the US Food and Drug Administration (FDA) for an additional indication for Nucala (mepolizumab) as an add-on treatment for severe eosinophilic asthma in paediatric patients aged six to 11 years.