Clinical Intelligence

Ophthotech announces results from third phase III trial of Fovist in Wet AMD

Tuesday, August 15, 2017

Ophthotech announced that the pre-specified primary endpoint of mean change in visual acuity at 12 months was not achieved in its phase III clinical trial investigating the superiority of Fovista (pegpleranib) anti-PDGF therapy in combination with Eylea (aflibercept) or Avastin (bevacizumab) anti-VEGF therapy compared to Eylea or Avastin monotherapy for the treatment of wet age-related macular degeneration (AMD). The addition of 1.5mg of Fovista to an Eylea or Avastin regimen did not result in benefit as measured by the mean change in visual acuity at the 12-month time point.

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Gilead announces FDA priority review designation for fixed-dose treatment of HIV

Friday, August 11, 2017

Gilead Sciences, Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted priority review for the company’s New Drug Application (NDA) for an investigational, fixed-dose combination of bictegravir (50mg) (BIC), a novel investigational integrase strand transfer inhibitor (INSTI) and emtricitabine/tenofovir alafenamide (200/25mg) (FTC/TAF), a dual-NRTI backbone, for the treatment of HIV-1 infection. Gilead filed the NDA for BIC/FTC/TAF with a Priority Review voucher on June 12, 2017, and the FDA has set a target action date under the Prescription Drug User Fee Act (PDUFA) of February 12, 2018.

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Ocugen receives FDA Orphan designation for ocular graft versus host disease

Thursday, August 10, 2017

Ocugen, a clinical stage biopharmaceutical company developing novel treatments for sight-threatening diseases, announced the FDA has granted orphan drug designation (ODD) for OCU300 (brimonidine tartrate) for the treatment of ocular graft versus host disease (oGVHD). Ocular GVHD is a common complication that occurs in 40-60% of patients who have undergone allergenic hematological stem cell transplantation (allo-SCT) or bone marrow transplants. Driven by autoimmune inflammation, oGVHD induces severe ocular surface disease, which over time significantly diminishes quality of life, and restricts daily activities due to visual impairment.

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AGTC files IND for X-linked retinitis pigmentosa treatment

Thursday, August 10, 2017

Applied Genetic Technologies, a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, has filed an Investigational New Drug application (IND) with the FDA to conduct a phase I/II clinical trial of the company’s gene therapy product candidate for the treatment of X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene.

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First FDA-approved treatment for ALS in 22 years now available in U.S.

Wednesday, August 9, 2017

Mitsubishi Tanabe Pharma America announced RADICAVA (edaravone), an intravenous therapy indicated for all adult patients diagnosed with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is now available for treatment in the U.S. RADICAVA, the first FDA-approved ALS treatment option in more than 20 years, has been demonstrated to slow the decline in the loss of physical function in ALS patients by 33 percent in its clinical trial.

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FDA approves Idhifa for relapsed or refractory acute myeloid leukemia

Wednesday, August 2, 2017

The FDA approved Idhifa (enasidenib) for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) who have a specific genetic mutation. The drug is approved for use with a companion diagnostic, the RealTime IDH2 Assay, which is used to detect specific mutations in the IDH2 gene in patients with AML.

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Acalabrutinib granted FDA Breakthrough Designation for MCL

Wednesday, August 2, 2017

AstraZeneca and its hematology research and development center of excellence, Acerta Pharma, announced that the FDA has granted Breakthrough Therapy Designation for acalabrutinib for the treatment of patients with mantle cell lymphoma (MCL) who have received at least one prior therapy. Acalabrutinib is a highly-selective, potent Bruton tyrosine kinase (BTK) inhibitor in development for the treatment of multiple B-cell cancers.

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