FDA News

Amicus to Seek Accelerated US and European Approval of its Investigational Fabry Disease Drug

Amicus Therapeutics announced its plans to file for accelerated approval of its investigational drug for a rare, inherited disease, following meetings with regulators in the US and Europe. The company said that it has recently met with regulatory authorities in Europe and the US to discuss the approval pathways for …

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With FDA’s Approval of Cholbam, Retrophin Gains Rare Disease Drug and Voucher

Orphan drug biotech Retrophin saw its shares go up nearly 30 percent today, after news hit that not only was the company’s rare disease drug approved, but that it also received a priority review voucher from US health regulators. Today, the US Food and Drug Administration (FDA) approved Cholbam (cholic …

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FDA Grants Orphan Drug Designation to Selten Pharma’s Pulmonary Arterial Hypertension Candidate

Today, privately-held, rare disease focused Selten Pharma, Inc. announced that US health regulators granted its investigational pulmonary arterial hypertension (PAH) drug orphan drug designation. The company said that its lead compound tacrolimus (SPI-026) has been granted orphan drug designation (ODD) by the US Food and Drug Administration (FDA) for the …

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Bristol-Myers Squibb’s Resubmitted Application for its Hepatitis C Drug Accepted by the FDA

The US Food and Drug Administration (FDA) has accepted for review Bristol-Myers Squibb’s (BMS) resubmitted application for its investigational hepatitis C drug. The company said that the agency accepted its New Drug Application (NDA) for daclatasvir, an investigational NS5A replication complex inhibitor, for use in combination with Gilead’s sofosbuvir for …

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FDA Approves United Therapeutics’ Rare Pediatric Cancer Drug and Gives the Company a Priority Review Voucher

US health regulators approved the first drug to treat pediatric patients with high-risk neuroblastoma, a rare cancer that develops on immature nerve cells. United Therapeutics Corporation announced that the US Food and Drug Administration (FDA) has approved Unituxin (dinutuximab) Injection, in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), interleukin-2 (IL-2) and …

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FDA Clears First-of-Kind Device to Treat Patients with Dialysis-Related Amyloidosis

US health regulators cleared use of a first-of-its-kind device to treat dialysis-related amyloidosis (DRA). The US Food and Drug Administration (FDA) authorized use of Lixelle Beta 2-microglobulin Apheresis Column under its pathway for medical devices that treat rare conditions. The device is used to treat DRA, a chronic, progressive condition …

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FDA Approves Astellas’ Cresemba for Rare but Serious Fungal Infections

The US Food and Drug Administration (FDA) has approved Astellas’ new antifungal drug to treat two rare, often fatal invasive fungal infections. The agency approved Astellas’ New Drug Application (NDA) for the use of Cresemba (isavuconazonium sulfate), the prodrug for isavuconazole, for patients 18 years of age and older in …

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FDA Approves Sandoz Zarxio as First Biosimilar in the US Market

Sandoz, a Novartis company, is the first company to receive approval of a biosimilar in the US through the new biosimilars pathway. The US Food and Drug Administration (FDA) approved Sandoz’s Zarxio (filgrastim-sndz) for all indications included in the label of the reference product, Neupogen. Amgen’s Neupogen (filgrastim) was originally …

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