Abeona Therapeutics Inc., of New York


Autologous, gene-corrected cell therapy

Recessive dystrophic epidermolysis bullosa

Received clinical hold letter from FDA, clarifying that agency will not provide approval for start of phase III until company submits additional data points on transport stability of EB-101 to clinical sites; Abeona anticipates receiving CMC clearance for planned Viital trial in the fourth quarter of 2019

Annexon Biosciences Inc., of South San Francisco


Monoclonal antibody

Guillain-Barré syndrome

FDA granted fast track designation

Astrazeneca plc, of Cambridge, U.K.

Qtrilmet (metformin hydrochloride, saxagliptin and dapagliflozin)

Modified-release tablets comprising an SGLT2 inhibitor, DPP-4 inhibitor and metformin extended-release

Type 2 diabetes

EMA's Committee for Medicinal Products for Human Use recommended approval

Avadel Pharmaceuticals plc, of Dublin


Once-nightly sodium oxybate


FDA agreed to proposed amendments to the statistical analysis plan and protocol under its SPA for the phase III study, resulting in lower sample size needed to show significance for both excessive daytime sleepiness and cataplexy; Rest-on trial now will enroll about 205 patients, with top-line data expected in the second quarter of 2020

Biogen Inc., of Cambridge, Mass.

Plegridy (peginterferon beta-1a) and Avonex (interferon beta-1a)

Interferon beta

Relapsing multiple sclerosis

EMA's Committee for Medicinal Products for Human Use recommended removing pregnancy contraindications to allow, where clinically needed, use during pregnancy and breastfeeding

Carsgen Therapeutics Inc., of Shanghai


Fully human anti-BCMA autologous CAR T cells

Relapsed or refractory multiple myeloma

EMA granted priority medicines, or PRIME, eligibility

Celltrion Healthcare Co. Ltd., of Incheon, South Korea


Subcutaneous version of infliximab biosimilar Remsima

Rheumatoid arthritis

EMA's Committee for Medicinal Products for Human Use recommended approval

GW Pharmaceuticals plc, London



Lennox-Gastaut syndrome or Dravet syndrome

Approved by European Commission for use as adjunctive therapy of seizures associated with LGS or Dravet, in conjunction with clobazam, for patients 2 and older

Mateon Therapeutics Inc., of Agoura Hills, Calif.


RNA therapeutic designed to abrogate immunosuppressive actions of TGF-beta 2

Diffuse intrinsic pontine glioma

FDA granted pediatric disease designation

Merck KGaA, of Darmstadt, Germany


Interferon beta-1a

Relapsing multiple sclerosis

EMA recommended updating product label to include that women may continue treatment during pregnancy if clinically needed and while breastfeeding

Phoenix Molecular Designs Ltd., of San Diego, and Vancouver, British Columbia


Oral RSK inhibitor

Triple-negative breast cancer

FDA cleared the IND for a phase I/Ib trial, set to start in the fourth quarter of 2019; trial will enroll patients with advanced disease and a subgroup of women with TNBC; study also will include a CAP/CLIA certified companion diagnostic designed to detect RSK2 activation

Rocket Pharmaceuticals Inc., of New York


Lentiviral vector-based gene therapy

Pyruvate kinase deficiency

The Spanish Agency for Medicines and Health Products cleared the firm to start an open-label, single-arm phase I trial in adult and pediatric transfusion-dependent PKD patients

Xynomic Pharmaceuticals Holdings Inc., of Shanghai


HDAC inhibitor

Relapsed or refractory follicular lymphoma

FDA granted fast track designation for use as a single agent, as a fourth-line treatment


For more information about individual companies and/or products, see Cortellis.


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