News

Feb 12 2019

Harmony Biosciences Announces File Acceptance Of Its New Drug Application For Pitolisant

Feb 12 2019

PLYMOUTH MEETING, Pa.Feb. 12, 2019 /PRNewswire/ — Harmony Biosciences, LLC (Harmony) announced today that the U.S. Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) for its investigational product, pitolisant, and has granted Priority Review for this NDA. Pitolisant is a first-in-class molecule with a novel mechanism of action; it is a potent and highly selective histamine 3 (H3) receptor antagonist/inverse agonist for the potential treatment of excessive daytime sleepiness (EDS) and/or cataplexy in adult patients with narcolepsy. A Priority Review designation by the FDA indicates that, if approved, pitolisant would provide a significant improvement in the safety or effectiveness of the treatment of EDS and/or cataplexy in adult patients with narcolepsy when compared to existing treatments. Harmony’s goal is to obtain FDA approval to market pitolisant in the U.S. in 2019.

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Jan 26 2019

BridgeBio Pharma Closes $299.2 Million Financing Round to Support Its Efforts to Target Genetic Disease at the Source

Jan 26 2019

PALO ALTO, Calif.–(BUSINESS WIRE)–BridgeBio Pharma, a clinical-stage biopharmaceutical company focused on genetic diseases, today announced a new financing round of $299.2 million. The round was co-led by existing investors KKR and Viking Global Investors. Other existing investors participating included Perceptive Advisors, AIG, Aisling Capital, Cormorant Capital, and Hercules Capital; and they were joined by new investors Sequoia Capital, and a blue-chip long-term investor. The financing will be used to support BridgeBio Pharma’s existing drug research and development programs and expand its efforts to rapidly develop medicines for patients with unmet needs.

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Jan 15 2019

TransEnterix Receives FDA 510(k) Clearance for Senhance Ultrasonic System

Jan 15 2019

RESEARCH TRIANGLE PARK, N.C.–(BUSINESS WIRE)–Jan. 15, 2019– TransEnterix, Inc. (NYSE American:TRXC), a medical device company that is digitizing the interface between surgeons and patients to improve minimally invasive surgery, today announced the Company received FDA 510(k) clearance for its Senhance™ Ultrasonic System.

“Advanced energy devices are used within a high percentage of cases across a wide range of procedures, which make them a critical tool for laparoscopic surgeons,” said Todd M. Pope, TransEnterix president and CEO. “We believe the addition of the Senhance Ultrasonic System is significant and broadens the attractiveness of the Senhance platform and digital laparoscopy for surgeons in the U.S.” Advanced energy devices, including ultrasonic devices, represent some of the most versatile and critical tools for surgeons in minimally invasive surgery. These instruments deliver controlled energy to effectively ligate and divide tissue, and minimize thermal injury to surrounding structures. The Company’s Senhance Ultrasonic System is now available in the U.S., as well as all countries that accept a CE Mark.

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Jan 7 2019

Lilly Announces Agreement To Acquire Loxo Oncology

Jan 7 2019

Eli Lilly and Company (NYSE: LLY) and Loxo Oncology, Inc. (NASDAQ: LOXO) today announced a definitive agreement for Lilly to acquire Loxo Oncology for $235.00 per share in cash, or approximately $8.0 billion. Loxo Oncology is a biopharmaceutical company focused on the development and commercialization of highly selective medicines for patients with genomically defined cancers.

The acquisition would be the largest and latest in a series of transactions Lilly has conducted to broaden its cancer treatment efforts with externally sourced opportunities for first-in-class and best-in-class therapies. Loxo Oncology is developing a pipeline of targeted medicines focused on cancers that are uniquely dependent on single gene abnormalities that can be detected by genomic testing. For patients with cancers that harbor these genomic alterations, a targeted medicine could have the potential to treat the cancer with dramatic effect.

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Dec 21 2018

Aimmune Therapeutics Submits BLA to FDA for AR101 for the Treatment of Peanut Allergy in Children and Adolescents Ages 4–17

Dec 21 2018

BRISBANE, Calif.–(BUSINESS WIRE)–Dec. 21, 2018– Aimmune Therapeutics, Inc. (Nasdaq: AIMT), a biopharmaceutical company developing treatments for potentially life-threatening food allergies, today announced that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for AR101, an investigational biologic oral immunotherapy for the treatment of peanut allergy in children and adolescents ages 4–17. The FDA granted AR101 Fast Track Designation for peanut allergy in September 2014 and Breakthrough Therapy Designation for peanut allergy in ages 4–17 in June 2015.

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Dec 19 2018

AVROBIO Receives Orphan-Drug Designation from the U.S. FDA for AVR-RD-01 for the Treatment of Fabry Disease

Dec 19 2018

CAMBRIDGE, Mass., Dec. 19, 2018 (GLOBE NEWSWIRE) — AVROBIO, Inc. (NASDAQ:AVRO) (the “Company”), a Phase 2 clinical-stage gene therapy company developing gene therapies to potentially cure rare diseases with a single dose, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for the Company’s investigational gene therapy, AVR-RD-01, for the treatment of Fabry disease. The gene therapy consists of the patient’s own hematopoietic stem cells transduced with AVROBIO’s lentiviral vector, carrying a functional version of the GLA gene that encodes α‑galactosidase A (AGA) – the enzyme that is deficient in Fabry disease. Patients with Fabry disease are currently enrolling in AVRO-RD-01-201, the Company’s Phase 2 clinical trial (FAB-201), as well as in an investigator-sponsored Phase 1 study.

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Dec 10 2018

Biohaven Announces Positive Results from Ongoing Rimegepant Long-Term Safety Study

Dec 10 2018

NEW HAVEN, Conn., Dec. 10, 2018 /PRNewswire/ — Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) today announced initial positive results from its ongoing long-term, open-label safety study of its oral calcitonin gene-related peptide (CGRP) receptor antagonist, rimegepant, for the acute treatment of migraine. Based upon this interim analysis (database cutoff of November 21, 2018) of Study BHV3000-201 (NCT03266588 or “Study 201”) it appears that rimegepant may be safe and well tolerated with long-term dosing in patients with migraine. In this study, patients are allowed to treat migraine attacks of all severities (mild to severe) up to once daily for up to a full year.

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Dec 3 2018

Biohaven Delivers Positive Phase 3 Results with Rimegepant Zydis® Orally Dissolving Tablet (ODT): Rapid and Lasting Benefit for the Acute Treatment of Migraine

Dec 3 2018

NEW HAVEN, Conn., Dec. 3, 2018 /PRNewswire/ — Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) today announced positive topline results from a randomized, controlled Phase 3 clinical trial (BHV3000-303 or Study 303) evaluating the efficacy and safety of its Zydis® orally dissolving tablet (ODT) formulation of rimegepant, an oral calcitonin gene-related peptide (CGRP) receptor antagonist, for the acute treatment of migraine. In Study 303, rimegepant Zydis ODT statistically differentiated from placebo on the two co-primary endpoints as well as the first 21 consecutive primary and secondary outcome measures that were prespecified in hierarchical testing. Consistent with the two previous Phase 3 clinical trials, Study 303 met its co-primary registrational endpoints of pain freedom and freedom from most bothersome symptom (MBS) at 2 hours using a single dose.

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Nov 26 2018

FDA Approves Vitrakvi® (larotrectinib), the First Ever TRK Inhibitor, for Patients with Advanced Solid Tumors Harboring an NTRK Gene Fusion

Nov 26 2018

STAMFORD, Conn. and WHIPPANY, N.J., Nov. 26, 2018 (GLOBE NEWSWIRE) — The U.S. Food and Drug Administration (FDA) today approved Vitrakvi® (larotrectinib), the first ever oral TRK inhibitor, for the treatment of adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have no satisfactory alternative treatments or that have progressed following treatment. This indication is approved under accelerated approval based on overall response rate (ORR) and duration of response (DOR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. Vitrakvi is the first treatment to receive a tumor-agnostic indication at the time of initial FDA approval. In clinical trials of patients with TRK fusion cancer, Vitrakvi demonstrated an ORR of 75 percent (N=55) (95% CI, 61%, 85%), including a 22 percent complete response (CR) rate.

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Nov 20 2018

LEO Pharma and PellePharm Announce $760 Million Collaboration to Advance Innovative Therapies for Rare Skin Diseases

Nov 20 2018

SAN FRANCISCO & BALLERUP, Denmark–(BUSINESS WIRE)–Danish dermatology specialists LEO Pharma and California rare disease pioneers PellePharm today announced a strategic development and commercialization collaboration to address unmet medical needs across various skin diseases with no approved treatments, advancing innovation and access to potential therapies for patients with life-altering conditions, such as Gorlin Syndrome and High Frequency Basal Cell Carcinoma (BCC), two distinct and rare forms of skin cancer.

Under the terms of the agreement, LEO Pharma has initially committed $70 million comprised of equity financing and financial R&D support to fund the global Phase 3 trial for patidegib topical gel 2% for the prevention and treatment of Gorlin Syndrome, with LEO Pharma securing an option to acquire all shares in PellePharm. PellePharm and its stockholders could receive up to an additional $690 million including merger consideration, and regulatory and commercial milestone payments. In addition, PellePharm stockholders are eligible to receive a double-digit royalty after achieving certain commercial milestones.

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