BioPharma Global

PRESS RELEASE: Pace® Life Sciences acquired BioPharma Global!

Read more about this exciting news here:
https://www.pacelabs.com/company/news-and-insights/life-sciences/pace-life-sciences-acquires-biopharma-global-expanding-fda-regulatory-affairs-strategy-and-consulting-capabilities-to-the-biotechnology-and-pharmaceutical-markets/

BridgeBio bounces back? Phase 2 readout provides hope for approval of drug to rebalance calcium levels

Read more: https://www.biopharmaglobal.com/2022/08/29/bridgebio-bounces-back-phase-2-readout-provides-hope-for-approval-of-drug-to-rebalance-calcium-levels/

BridgeBio bounces back? Phase 2 readout provides hope for approval of drug to rebalance calcium levels After a series of unfortunate events for BridgeBio Pharma, the biotech may be on a new upward trajectory as it unveiled positive data from 13 patients taking a drug to treat low calcium levels.

Bluebird Bio gene therapy wins first FDA approval for rare blood disorder

Read more: https://www.biopharmaglobal.com/2022/08/26/bluebird-bio-gene-therapy-wins-first-fda-approval-for-rare-blood-disorder/

Bluebird Bio gene therapy wins first FDA approval for rare blood disorder SAN FRANCISCO, Aug. 08, 2022 (GLOBE NEWSWIRE) -- Attralus, Inc., a clinical stage biopharmaceutical company developing transformative medicines to improve the lives of patients with systemic amyloidosis, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designa...

This Week's Rare Disease Highlight is Stargardt Disease

Read more: https://www.biopharmaglobal.com/2022/08/25/rare-disease-highlight-stargardt-disease/

Rare Disease Highlight: Stargardt Disease - BioPharma Global Stargardt Disease, also known as juvenile macular degeneration, is a rare disease of the eyes in which part of the eye called the retina does not function correctly. This is the most common form of inherited macular degeneration, affecting about 30,000 people in the United States2. Stargardt’s is ...

Attralus Receives U.S. FDA Orphan Drug Designation for AT-01 (Iodine (I-124) Evuzamitide), an Investigational Diagnostic for the Management of Transthyretin Amyloidosis

Read more: https://www.biopharmaglobal.com/2022/08/24/attralus-receives-u-s-fda-orphan-drug-designation-for-at-01-iodine-i-124-evuzamitide-an-investigational-diagnostic-for-the-management-of-transthyretin-amyloidosis/

Attralus Receives U.S. FDA Orphan Drug Designation for AT-01 (Iodine (I-124) Evuzamitide), an Investigational Diagnostic for the Management of Transthyretin Amyloidosis SAN FRANCISCO, Aug. 08, 2022 (GLOBE NEWSWIRE) -- Attralus, Inc., a clinical stage biopharmaceutical company developing transformative medicines to improve the lives of patients with systemic amyloidosis, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designa...

ALX Oncology’s Evorpacept Receives Fast Track Designation from FDA as First-Line Treatment for Head and Neck Squamous Cell Carcinoma

Read more: https://www.biopharmaglobal.com/2022/08/22/alx-oncologys-evorpacept-receives-fast-track-designation-from-fda-as-first-line-treatment-for-head-and-neck-squamous-cell-carcinoma/

ALX Oncology’s Evorpacept Receives Fast Track Designation from FDA as First-Line Treatment for Head and Neck Squamous Cell Carcinoma SOUTH SAN FRANCISCO, Calif., Aug. 01, 2022 (GLOBE NEWSWIRE) -- ALX Oncology Holdings Inc., (“ALX Oncology”) (Nasdaq: ALXO) a clinical-stage immuno-oncology company developing therapies that block the CD47 checkpoint pathway, today announced that the U.S. Food and Drug Administration (“FDA”) ...

FDA Accepts Biogen’s New Drug Application and Grants Priority Review of Tofersen for a Rare, Genetic Form of ALS

Read more: https://www.biopharmaglobal.com/2022/08/19/fda-accepts-biogens-new-drug-application-and-grants-priority-review-of-tofersen-for-a-rare-genetic-form-of-als/

FDA Accepts Biogen’s New Drug Application and Grants Priority Review of Tofersen for a Rare, Genetic Form of ALS CAMBRIDGE, Mass., July 26, 2022 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for tofersen, an investigational drug for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). The a...

This Week's Rare Disease Highlight is Netherton Syndrome

Read more: https://www.biopharmaglobal.com/2022/08/18/rare-disease-highlight-netherton-syndrome/

Rare Disease Highlight: Netherton Syndrome - BioPharma Global Netherton syndrome (NS), also referred to as bamboo hair syndrome, is a rare and severe genetic skin disorder [1-3]. The disease is characterized by chronic ichthyosiform erythroderma (a condition of fine white and red scales across the entire body), atopy (a genetic tendency to develop allergic dis...

FDA, in another test of its flexibility, agrees to review Biogen’s closely watched ALS drug

Read more: https://www.biopharmaglobal.com/2022/08/17/fda-in-another-test-of-its-flexibility-agrees-to-review-biogens-closely-watched-als-drug/

FDA, in another test of its flexibility, agrees to review Biogen’s closely watched ALS drug Late last year, Biogen disclosed results from a closely watched study evaluating an experimental drug for Lou Gehrig’s disease, also known as ALS or amyotrophic lateral sclerosis. They showed the drug, called tofersen, wasn’t better than a placebo at slowing progression of the fatal nerve disord...

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Ionis announces that FDA accepts New Drug Application and grants Priority Review of tofersen for a rare, genetic form of ALS

Read more: https://www.biopharmaglobal.com/2022/08/15/ionis-announces-that-fda-accepts-new-drug-application-and-grants-priority-review-of-tofersen-for-a-rare-genetic-form-of-als/

Ionis announces that FDA accepts New Drug Application and grants Priority Review of tofersen for a rare, genetic form of ALS CARLSBAD, Calif., July 26, 2022 /PRNewswire/ -- Ionis Pharmaceuticals (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for tofersen, an investigational antisense medicine for the treatment of superoxide dismutase 1 amyotrophic....

Pliant Therapeutics Receives FDA Fast Track Designation for PLN-74809 for the Treatment of Primary Sclerosing Cholangitis

Read more: https://www.biopharmaglobal.com/2022/08/12/pliant-therapeutics-receives-fda-fast-track-designation-for-pln-74809-for-the-treatment-of-primary-sclerosing-cholangitis/

Pliant Therapeutics Receives FDA Fast Track Designation for PLN-74809 for the Treatment of Primary Sclerosing Cholangitis SOUTH SAN FRANCISCO, Calif., July 21, 2022 (GLOBE NEWSWIRE) -- Pliant Therapeutics (NASDAQ: PLRX) announced today that PLN-74809, its oral, dual-selective αvß6 / αvß1 integrin inhibitor, has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for the potential treatm...

Wugen Receives U.S. FDA Fast Track and Rare Pediatric Disease Designations for WU-CART-007 for the Treatment of R/R T-ALL/LBL

Read more: https://www.biopharmaglobal.com/2022/08/10/wugen-receives-u-s-fda-fast-track-and-rare-pediatric-disease-designations-for-wu-cart-007-for-the-treatment-of-r-r-t-all-lbl/

Wugen Receives U.S. FDA Fast Track and Rare Pediatric Disease Designations for WU-CART-007 for the Treatment of R/R T-ALL/LBL ST. LOUIS & SAN DIEGO--(BUSINESS WIRE)--Wugen, Inc., a clinical-stage biotechnology company developing a pipeline of allogeneic cell therapies to treat a broad range of hematological and solid tumor malignancies, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track...

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AVROBIO Receives Orphan Drug Designation from the U.S. Food and Drug Administration for AVR-RD-05, a Gene Therapy for Mucopolysaccharidosis Type II (MPSII) or Hunter Syndrome

Read more: https://www.biopharmaglobal.com/2022/08/08/avrobio-receives-orphan-drug-designation-from-the-u-s-food-and-drug-administration-for-avr-rd-05-a-gene-therapy-for-mucopolysaccharidosis-type-ii-mpsii-or-hunter-syndrome/

AVROBIO Receives Orphan Drug Designation from the U.S. Food and Drug Administration for AVR-RD-05, a Gene Therapy for Mucopolysaccharidosis Type II (MPSII) or Hunter Syndrome CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for AVR-RD-05,....

Anthos Therapeutics Announces that Abelacimab has Received FDA Fast Track Designation for the Treatment of Thrombosis Associated with Cancer

Read more: https://www.biopharmaglobal.com/2022/08/05/anthos-therapeutics-announces-that-abelacimab-has-received-fda-fast-track-designation-for-the-treatment-of-thrombosis-associated-with-cancer/

Anthos Therapeutics Announces that Abelacimab has Received FDA Fast Track Designation for the Treatment of Thrombosis Associated with Cancer CAMBRIDGE, Mass., July 11, 2022 /PRNewswire/ -- Anthos Therapeutics, a clinical-stage biotechnology company developing innovative therapies for cardiovascular and metabolic diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its investigat...

This Week's Rare Disease Highlight is Alternating Hemiplegia of Childhood

Read more: https://www.biopharmaglobal.com/2022/08/04/rare-disease-highlight-alternating-hemiplegia-of-childhood/

Rare Disease Highlight: Alternating Hemiplegia of Childhood - BioPharma Global Alternating Hemiplegia of Childhood (AHC) is a rare and severe neurodevelopmental disorder that fully manifests during early childhood, typically within the first year of life. AHC is characterized by repeated attacks of hemiplegia, paralysis of one side of the body, or episodes of quadriplegia, sim...

Phanes Therapeutics' PT886 granted Orphan Drug Designation for the treatment of pancreatic cancer by the FDA

Read more: https://www.biopharmaglobal.com/2022/08/03/phanes-therapeutics-pt886-granted-orphan-drug-designation-for-the-treatment-of-pancreatic-cancer-by-the-fda/

Phanes Therapeutics' PT886 granted Orphan Drug Designation for the treatment of pancreatic cancer by the FDA SAN DIEGO, June 30, 2022 /PRNewswire/ -- Phanes Therapeutics, Inc. (Phanes), an emerging leader in innovative discovery research and clinical development in oncology, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to PT886 for the treatment of pa...

Aura Biosciences Receives FDA Fast Track Designation for Belzupacap Sarotalocan (AU-011) for the Treatment of Non-Muscle Invasive Bladder Cancer

Read more: https://www.biopharmaglobal.com/2022/08/01/aura-biosciences-receives-fda-fast-track-designation-for-belzupacap-sarotalocan-au-011-for-the-treatment-of-non-muscle-invasive-bladder-cancer/

Aura Biosciences Receives FDA Fast Track Designation for Belzupacap Sarotalocan (AU-011) for the Treatment of Non-Muscle Invasive Bladder Cancer CAMBRIDGE, Mass.--(BUSINESS WIRE)--Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast....

ALX Oncology Receives U.S. FDA Orphan Drug Designation for Evorpacept for the Treatment of Patients with Acute Myeloid Leukemia

Read more:https://www.biopharmaglobal.com/2022/07/29/alx-oncology-receives-u-s-fda-orphan-drug-designation-for-evorpacept-for-the-treatment-of-patients-with-acute-myeloid-leukemia/

ALX Oncology Receives U.S. FDA Orphan Drug Designation for Evorpacept for the Treatment of Patients with Acute Myeloid Leukemia SOUTH SAN FRANCISCO, Calif., June 29, 2022 (GLOBE NEWSWIRE) -- ALX Oncology Holdings Inc., (“ALX Oncology”) (Nasdaq: ALXO) a clinical-stage immuno-oncology company developing therapies that block the CD47 checkpoint pathway, today announced that the U.S. Food and Drug Administration (“FDA”) ...

Apnimed Granted FDA Fast Track Designation for AD109, a Novel First-in-Class Oral Pharmacologic Combination for the Treatment of Obstructive Sleep Apnea (OSA)

Read more: https://www.biopharmaglobal.com/2022/07/27/apnimed-granted-fda-fast-track-designation-for-ad109-a-novel-first-in-class-oral-pharmacologic-combination-for-the-treatment-of-obstructive-sleep-apnea-osa/

Apnimed Granted FDA Fast Track Designation for AD109, a Novel First-in-Class Oral Pharmacologic Combination for the Treatment of Obstructive Sleep Apnea (OSA) Apnimed, a clinical-stage pharmaceutical company focused on developing oral pharmacologic treatments to address obstructive sleep apnea (OSA) and related disorders, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its oral pharmacologic AD109 fo...

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BioMarin Receives Positive CHMP Opinion in Europe for Valoctocogene Roxaparvovec Gene Therapy to Treat Adults with Severe Hemophilia A

Read more: https://www.biopharmaglobal.com/2022/07/25/biomarin-receives-positive-chmp-opinion-in-europe-for-valoctocogene-roxaparvovec-gene-therapy-to-treat-adults-with-severe-hemophilia-a/

BioMarin Receives Positive CHMP Opinion in Europe for Valoctocogene Roxaparvovec Gene Therapy to Treat Adults with Severe Hemophilia A SAN RAFAEL, Calif., June 24, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending conditional marketing authorization (CMA) for its investigational gene therapy, val...

Adverum Biotechnologies Granted Priority Medicines (PRIME) Designation by European Medicines Agency for ADVM-022 in Wet AMD

Read more: https://www.biopharmaglobal.com/2022/07/22/adverum-biotechnologies-granted-priority-medicines-prime-designation-by-european-medicines-agency-for-advm-022-in-wet-amd/

Adverum Biotechnologies Granted Priority Medicines (PRIME) Designation by European Medicines Agency for ADVM-022 in Wet AMD REDWOOD CITY, Calif., June 24, 2022 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced that the European Medicines Agency (EMA) has granted ADVM-022 Priority Medicines (PRI...

This Week's Rare Disease Highlight is on Buruli Ulcer

Read more: https://www.biopharmaglobal.com/2022/07/21/rare-disease-highlight-buruli-ulcer/

Rare Disease Highlight: Buruli Ulcer - BioPharma Global Buruli ulcer (BU) is a chronic skin infection caused by Mycobacterium ulcerans (M. ulcerans) resulting in death of body tissue, also referred to as necrosis (Scherr et al., 2018). M. ulcerans is the third most common mycobacterial infection worldwide and is classified as a neglected tropical disease...

Regulus Therapeutics Announces Receipt of FDA Orphan Drug Designation for RGLS8429 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Read more: https://www.biopharmaglobal.com/2022/07/20/regulus-therapeutics-announces-receipt-of-fda-orphan-drug-designation-for-rgls8429-for-the-treatment-of-autosomal-dominant-polycystic-kidney-disease-adpkd/

Regulus Therapeutics Announces Receipt of FDA Orphan Drug Designation for RGLS8429 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) "Patients living with ADPKD currently have limited treatment options and approximately half of patients develop end-stage renal disease by age 60 requiring dialysis or transplantation," commented Jay Hagan, CEO of Regulus Therapeutics. "ADPKD is a disease of high unmet need affecting nearly 160,000....