Vicore’s lung disease digital therapy gets breakthrough tag
pharmaphorum
MARCH 20, 2024
Vicore gets FDA breakthrough status for Almee, a cognitive behavioural therapy (CBT) digital health therapy used to support people with pulmonary fibrosis
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After filing hitch, Eisai and Biogen begin rolling FDA submission for subcutaneous Leqembi
Fierce Pharma
MAY 14, 2024
regulators last month, Eisai and Biogen’s effort to deploy a more convenient version of their Alzheimer’s disease drug Leqembi appears to be back on track. After suffering a setback with U.S.
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Orchard’s MLD gene therapy becomes costliest US medicine
pharmaphorum
MARCH 20, 2024
Orchard Therapeutics has revealed the US price of Lenmeldy, its gene therapy for rare disease MLD, placing a $4.25m price tag on the one-shot treatment
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Not covering emerging Alzheimer's drugs could cost Medicare billions: study
Fierce Healthcare
JUNE 9, 2023
Emerging Alzheimer’s disease medications come with high price tags, but researchers with the University of Chicago calculate that in the long run, it’s much less costly to cover these therapies for | Emerging Alzheimer’s medications come with high price tags, but researchers with the University of Chicago calculate that in the long run, it’s much less (..)
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STAT+: Colorado lawmakers revise effort to exempt rare disease drugs from state cost caps
STAT
APRIL 12, 2024
In response to controversy, a group of Colorado lawmakers is abandoning a bill that would have required a new state board — which is chartered with setting caps for the cost of medicines — to create exemptions for rare disease drugs.
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NorthSea Therapeutics bags FDA rare paediatric disease tag for NASH drug
Pharmaceutical Technology
OCTOBER 17, 2023
The Netherlands-based company’s treatment has been awarded the designation by the US FDA following a successful Phase I trial.
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Skin tag on eyelid: Causes and treatment
The Checkup by Singlecare
FEBRUARY 17, 2024
Skin tags , also called acrochordons, are harmless growths that can occur anywhere on your body but tend to develop in more friction-heavy areas like the armpit, the neck along the seat belt line where clothes and jewelry touch, under the breasts, in the groin, or on the face—specifically, the eyelid. What causes skin tags on eyelids?
STAT+: CMS will use outcomes-based agreements in bid to help Medicaid pay for sickle cell gene therapies
STAT
JANUARY 30, 2024
In response to concerns over multimillion-dollar price tags for new gene therapies for sickle cell disease, the U.S. Sickle cell disease affects an estimated 100,000 Americans. Estimates vary, but the federal government believes as many as 60% of people with sickle cell disease rely on Medicaid for health care coverage.
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ICER updates cost-effectiveness threshold for bluebird, Vertex and CRISPR's dueling sickle cell gene therapies
Fierce Pharma
JULY 14, 2023
The potentially curative promise of gene therapies often carries a steep price tag. | The potentially curative promise of gene therapies often carries a steep price tag. But for a pair of personalized medicine prospects in sickle cell disease (SCD), the cost could be worth it, at least as far as ICER is concerned.
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Revolutionizing Rare Disease Treatment: Embracing the Patient-Centric Transformation in Pharmaceutical Customer Engagement
Pharmaceutical Commerce
MARCH 6, 2024
Navigating the complex landscape of healthcare coverage can be an intimidating task, especially for patients with rare diseases for which treatments often come with a high price tag.
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Bluebird won't play off Vertex in deciding price for its lovo-cel gene therapy, exec says
Fierce Pharma
NOVEMBER 7, 2023
Two long-awaited treatments for sickle cell disease (SCD) are on the docket for FDA decisions next month. Two long-awaited treatments for sickle cell disease (SCD) are on the docket for FDA decisions next month. Vertex and CRISPR Therapeutics are up first with a Dec. |
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Opinion: Sickle cell cures are coming. African children can’t be left behind
STAT
JULY 12, 2023
The treatment of sickle cell disease is on the cusp of a historic breakthrough, with makers of two gene-based treatments for the debilitating blood disorder hoping for regulatory approval this year. Read the rest…
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PharmaShots Weekly Snapshots (April 03 - 07, 2023)
PharmaShots
APRIL 7, 2023
TG Therapeutics Receives EMA’s CHMP Positive Opinion of Briumvi (ublituximab-xiiy) for the Treatment of Relapsing Forms of Multiple Sclerosis Date: Apr 3, 2023 | Tags: TG Therapeutics, Briumvi, Ublituximab-xiiy, Multiple Sclerosis, Regulatory, EMA, CHMP BMS Receives EMA’s CHMP Positive Opinion of Breyanzi (lisocabtagene maraleucel) for (..)
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PharmaShots Weekly Snapshots (March 20 - 24, 2023)
PharmaShots
MARCH 24, 2023
PTC Therapeutics’ Upstaza (eladocagene exuparvovec) Receives NICE Recommendation for the Treatment of AADC Deficiency Date: Mar 24, 2023 | Tags: PTC Therapeutics, Upstaza, eladocagene exuparvovec, AADC Deficiency, Regulatory, NICE Onconova Therapeutics Entered into a Research Collaboration with Pangea Biomed to Identify Biomarkers for Cancer (..)
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STAT+: Kyowa Kirin to acquire Orchard Therapeutics, maker of gene therapy Libmeldy
STAT
OCTOBER 5, 2023
The medicines, which deliver functional genes to patients to overcome their genetic diseases, are one-time treatments that can have profound effects for patients but that typically carry seven-figure price tags, leading to so far slow rollouts. Continue to STAT+ to read the full story…
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PharmaShots Weekly Snapshots (June 19 – 23, 2023)
PharmaShots
JUNE 23, 2023
Gilead Receives EMA’s CHMP Positive Opinion of Trodelvy (sacituzumab govitecan) for Pre-Treated HR+/HER2- Metastatic Breast Cancer Date: June 23, 2023 | Tags: Gilead, Trodelvy, sacituzumab govitecan, HR+/HER2- Metastatic Breast Cancer, Regulatory, EMA, CHMP, Positive Opinion Sarepta Therapeutics’ Elevidys Receives the US FDA’s Accelerated (..)
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PharmaShots Weekly Snapshots (May 01 - 05, 2023)
PharmaShots
MAY 5, 2023
Chiesi Global Rare Diseases and Protalix BioTherapeutics Receive EC’s Marketing Authorization of PRX-102 (pegunigalsidase alfa) for the Treatment of Fabry Disease Date: May 05, 2023 | Tags: Chiesi Global Rare Diseases, Protalix BioTherapeutics, PRX-102, pegunigalsidase alfa, Fabry Disease, Regulatory, EC, Marketing Authorization Kinoxis Therapeutics (..)
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PharmaShots Weekly Snapshots (May 15 - 19, 2023)
PharmaShots
MAY 19, 2023
Alimera Acquires Rights from EyePoint Pharmaceuticals to Commercialise Yutiq in the US Date: May 19, 2023 | Tags: Alimera, EyePoint Pharmaceuticals, Yutiq, Iluvien, chronic non-infectious uveitis, US, Pharma Sobi Reports EMA’s Validation of MAA for Efanesoctocog Alfa to Treat Haemophilia A Date: May 19, 2023 | Tags: Sobi, Efanesoctocog Alfa, (..)
PharmaShots Weekly Snapshots (May 22 - 26, 2023)
PharmaShots
MAY 26, 2023
Takeda and HUTCHMED's Fruquintinib Receives Priority Review from the US FDA to Treat Metastatic Colorectal Cancer Date: May 26, 2023 | Tags: Takeda, HUTCHMED, Fruquintinib, Metastatic Colorectal Cancer, Regulatory, Priority Review, US, FDA Gilead Receives EMA’s CHMP Positive Opinion to Extend the Use of Veklury (remdesivir) for COVID-19 Date: (..)
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PharmaShots Weekly Snapshots (June 05 – 09, 2023)
PharmaShots
JUNE 9, 2023
Quell Therapeutics Signed an Exclusive Option and License Agreement with AstraZeneca to Develop, Manufacture and Commercialize Engineered Treg Cell Therapies Date: June 09, 2023 | Tags: Quell Therapeutics, AstraZeneca, Engineered Treg Cell Therapies, Type 1 Diabetes, Inflammatory Bowel Disease, Biotech, Treg cell engineering modules Astellas and the (..)
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bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies
Pharmaceutical Technology
SEPTEMBER 30, 2022
Skysona is indicated as a one-time gene therapy to slow the progression of cerebral adrenoleukodystrophy (CALD), a rare paediatric neurodegenerative disease in boys aged 4–17 years diagnosed with early-stage CALD. These approvals represent crucial milestones for bluebird bio, the gene therapy field, and patients with rare genetic diseases.
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PharmaShots Weekly Snapshots (May 08 - 12, 2023)
PharmaShots
MAY 12, 2023
Sanofi Reports P-IIIb Trial (HARMONIE) Results of Nirsevimab for the Prevention of Hospitalizations due to RSV-Related LRTD Date: May 12, 2023 | Tags: Sanofi, Nirsevimab, RSV-Related LRTD, Clinical Trial, P-IIIb, HARMONIE Trial G1 Therapeutics Presents Preliminary Results from P-II Trial of Trilaciclib for Triple-Negative Breast Cancer at ESMO 2023 (..)
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40 PharmaShots Weekly Snapshots (April 24 - 28, 2023)
PharmaShots
APRIL 28, 2023
Difficile Infection Date: Apr 27, 2023 | Tags: Seres Therapeutics, Nestlé Health Science, Vowst, C.
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PharmaShots Weekly Snapshots (April 10 - 14, 2023)
PharmaShots
APRIL 14, 2023
Ocugen Reports Preliminary Results from the P-I/II Trial of OCU400 for the Treatment of Retinitis Pigmentosa and Leber Congenital Amaurosis Date: Apr 14, 2023 | Tags: Ocugen, OCU400, Retinitis Pigmentosa, Leber Congenital Amaurosis, Clinical Trial, P-I/II Trial Candesant Biomedical Receives the US FDA Clearance of Brella SweatControl Patch for Primary (..)
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STAT+: Bone marrow transplant advance could widen use of this less buzzy cure for sickle cell disease
STAT
DECEMBER 12, 2023
SAN DIEGO — The sickle cell community has for the past few days been buzzing with news of the first-ever approved gene therapies for the devastating disease. These drugs have price tags of $2.2 million and $3.1 million, respectively.
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PharmaShots Weekly Snapshots (February 20 - 24, 2023)
PharmaShots
FEBRUARY 24, 2023
Positive Solid Tumors Date: Feb 23, 2023 | Tags: Keymed, Lepu Biopharma, AstraZeneca, CMG901, Claudin 18.2-Positive Positive Solid Tumors Date: Feb 23, 2023 | Tags: Keymed, Lepu Biopharma, AstraZeneca, CMG901, Claudin 18.2-Positive Positive Solid Tumors Date: Feb 23, 2023 | Tags: Keymed, Lepu Biopharma, AstraZeneca, CMG901, Claudin 18.2-Positive
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PharmaShots Weekly Snapshots (March 27 - 31, 2023)
PharmaShots
MARCH 31, 2023
PDS Biotech to Initiate P-III Trial (VERSATILE-003) of PDS0101 + Keytruda (pembrolizumab) for Head and Neck Cancer Date: Mar 31, 2023 | Tags: PDS Biotech, PDS0101, Keytruda, pembrolizumab, Head, Neck Cancer, Clinical Trial, P-III, VERSATILE-003 Trial Omega Therapeutics Entered into a Clinical Supply Agreement with Roche to Evaluate OTX-2002 for Hepatocellular (..)
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40 PharmaShots Weekly Snapshots (February 06 - 10, 2023)
PharmaShots
FEBRUARY 10, 2023
LEO Pharma Reports P-III Trial (DELTA 2) Results of Delgocitinib for Chronic Hand Eczema Date: Feb 10, 2023 | Tags: LEO Pharma, Delgocitinib, Chronic Hand Eczema, Clinical Trial, P-III, DELTA 2 Trial GSK’s Jemperli (dostarlimab-gxly) Receives the US FDA’s Regular Approval for Recurrent or Advanced Mismatch Repair-Deficient Endometrial Cancer (..)
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PharmaShots Weekly Snapshots (February 06 - 10, 2023)
PharmaShots
FEBRUARY 10, 2023
LEO Pharma Reports P-III Trial (DELTA 2) Results of Delgocitinib for Chronic Hand Eczema Date: Feb 10, 2023 | Tags: LEO Pharma, Delgocitinib, Chronic Hand Eczema, Clinical Trial, P-III, DELTA 2 Trial GSK’s Jemperli (dostarlimab-gxly) Receives the US FDA’s Regular Approval for Recurrent or Advanced Mismatch Repair-Deficient Endometrial Cancer (..)
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STAT+: Pharmalittle: We’re reading about Wegovy supplies, Biogen ditching Aduhelm, and more
STAT
JANUARY 31, 2024
In response to concerns over multi-million-dollar price tags for new gene therapies for sickle cell disease, the U.S. Sickle cell disease affects an estimated 100,000 Americans. Estimates vary, but the federal government believes as many as 60% of people with sickle cell disease rely on Medicaid for health care coverage.
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PharmaShots Weekly Snapshots (June 12 – 16, 2023)
PharmaShots
JUNE 16, 2023
Date: June 12, 2023 | Tags: Novartis, Chinook Therapeutics, Atrasentan, Zigakibart, BION-1301, IgA nephropathy, M&A, ~$3.5B Date: June 12, 2023 | Tags: Novartis, Chinook Therapeutics, Atrasentan, Zigakibart, BION-1301, IgA nephropathy, M&A, ~$3.5B
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40 PharmaShots Weekly Snapshots (January 23 - 27, 2023)
PharmaShots
JANUARY 27, 2023
Magenta Therapeutics Pauses the P-I/II Study in AML Patients Date: Jan 27, 2023 | Tags: Magenta Therapeutics, MGTA-117, AML, Clinical Trial, P-I/II Ipsen Receives CHMP Negative Opinion for Palovarotene to Treat Fibrodysplasia Ossificans Progressiva Date: Jan 27, 2023 | Tags: Ipsen, Palovarotene, Fibrodysplasia Ossificans Progressiva, Regulatory, CHMP, (..)
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40 PharmaShots Weekly Snapshots (March 13 - 17, 2023)
PharmaShots
MARCH 17, 2023
5-Adapted Bivalent Booster to Treat COVID-19 in Children ≤5 Years Date: Mar 15, 2023 | Tags: Pfizer, BioNTech, Omicron BA.4/BA.5-Adapted 5-Adapted Bivalent Booster to Treat COVID-19 in Children ≤5 Years Date: Mar 15, 2023 | Tags: Pfizer, BioNTech, Omicron BA.4/BA.5-Adapted
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PharmaShots Weekly Snapshots (April 17 - 21, 2023)
PharmaShots
APRIL 21, 2023
Oblato Reports the First Patient Enrolment of OKN-007 in the P-I Clinical Trial for Recurrent High-Grade Glioma Date: Apr 21, 2023 | Tags: Oblato, OKN-007, Recurrent High-Grade Glioma, Regulatory, Henry Ford Health System CARsgen's CT041 Receives the US FDA’s IND Clearance for the Postoperative Adjuvant Therapy of Pancreatic Cancer Date: (..)
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PharmaShots Weekly Snapshots (February 13 - 17, 2023)
PharmaShots
FEBRUARY 17, 2023
Iveric Bio Reports the US FDA Acceptance of NDA and Granted Priority Review of Avacincaptad Pegol for Geographic Atrophy Date: Feb 17, 2023 | Tags: Iveric Bio, Avacincaptad Pegol, Geographic Atrophy, Regulatory, US, FDA, NDA, Priority Review ALX Oncology Reports the First Patient Dosing of Evorpacept in the P-I Study (ASPEN-07) for the Treatment of (..)
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PharmaShots Weekly Snapshots (May 29 - June 02, 2023)
PharmaShots
JUNE 2, 2023
Impact Therapeutics Entered into a License and Collaboration Agreement with Eikon Therapeutics to Develop and Commercialize IMP1734 Date: June 02, 2023 | Tags: Impact Therapeutics, Eikon Therapeutics, IMP1734, Pharma, China, Hong Kong, Macau, Taiwan BMS’ Mavacamten Receives the NICE Recommendation for the Treatment of Obstructive Hypertrophic (..)
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PharmaShots Weekly Snapshots (March 20 - 24, 2023)
PharmaShots
MARCH 24, 2023
PTC Therapeutics’ Upstaza (eladocagene exuparvovec) Receives NICE Recommendation for the Treatment of AADC Deficiency Date: Mar 24, 2023 | Tags: PTC Therapeutics, Upstaza, eladocagene exuparvovec, AADC Deficiency, Regulatory, NICE Onconova Therapeutics Entered into a Research Collaboration with Pangea Biomed to Identify Biomarkers for Cancer (..)
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40 PharmaShots Weekly Snapshots (January 30 - February 03, 2023)
PharmaShots
FEBRUARY 3, 2023
4D Molecular Therapeutics Receives the US FDA’s IND Clearance of 4D-150 for the Treatment of Diabetic Macular Edema Date: Feb 03, 2023 | Tags: 4D Molecular Therapeutics, 4D-150, Diabetic Macular Edema, Regulatory, US, FDA, IND AstraZeneca and Amgen Receive the US FDA’s Approval of Tezspire (tezepelumab) for the Treatment of Severe Asthma (..)
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Cost of disease-modifying therapies for multiple sclerosis continues to rise in US
Pharmaceutical Technology
MAY 19, 2023
Multiple sclerosis (MS) is a primary autoimmune disease in which inflammation is a core contributor to the degeneration of the central nervous system (CNS), leading to neurological disability and affecting sensory, visual, motor, and autonomic systems. DMTs for MS have a high price tag, particularly in the US.
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STAT+: Pharmalittle: Vertex exec says prices for gene therapies are ‘in the ballpark’; insulin access in poor countries remains spotty
STAT
OCTOBER 6, 2022
… Multimillion-dollar prices for a rival’s treatments with the potential to cure rare diseases are “in the right ballpark,” according to Stuart Arbuckle, chief operating officer at Vertex Pharmaceuticals, which is preparing to market its own treatment for sickle-cell disease , Bloomberg News tells us. Even
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Biogen, Eisai Alzheimer’s drug lecanemab fast tracked by FDA
pharmaphorum
DECEMBER 24, 2021
The anti-amyloid drug, which also claimed a breakthrough tag from the FDA in July, is being developed for the treatment of early-stage Alzheimer’s disease. The $56,000-a-year price tag for the drug, which has now been cut in half in the US, also hasn’t helped the drugmakers make a case for Aduhelm with US payers.
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5 Ways Your Pharmacy Can Participate in Parkinson’s Awareness Month
Digital Pharmacist
MARCH 28, 2024
April is the month of Parkinson’s Awareness and it is imperative we do our best to spread awareness for the more than 1 million Americans living with the disease. Each alphabet letter stands for an aspect of the disease to educate the population on the symptoms of PD and how to address them. Check out the ABCs of PD here.
BD partners with Biocorp on connected self-injection devices
pharmaphorum
OCTOBER 18, 2022
Medtech giant Beckton Dickinson (BD) has signed a deal with France’s Biocorp to use the latter’s near-field communication (NFC) tags in injectable devices. The Injay tag can confirm a complete injection and transfer that information via an NFC reader to a smartphone or tablet for review by a healthcare professional.
5 Things I Love About The MAD-ID Conference, The Antimicrobial Stewardship Meeting
IDStewardship
MAY 13, 2023
In this article an infectious diseases pharmacists discusses the MAD-ID conference, which is known as The Antimicrobial Stewardship Meeting, Making A Difference In Infectious Diseases (MAD-ID). Scroll to the bottom of this post for a list of infectious diseases meetings that have been sourced from Twitter. Gauthier, Pharm.D.,
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