PDUFA

Autolus Therapeutics presents longer-term follow-up and additional data analysis of Pivotal Phase 2 FELIX study of obe-cel for adult r/r B-ALL in an oral presentation at ASCO

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пятница, мая 31, 2024
Patient, Conference call, Survival, PIN, Haematopoietic system, Risk, BST, EFS, FDA, Therapy, Conference, PDUFA, MRD, Death, ALL, ASCO, SCT, Data analysis, OS, BLA, Society, Myelodysplastic syndrome, Webcast, Annual general meeting

At the February 7, 2024, data cut-off date, the majority of ongoing responders showed durable responses.

Key Points: 
  • At the February 7, 2024, data cut-off date, the majority of ongoing responders showed durable responses.
  • Patients who experienced B-cell recovery had a 1.7 fold increased risk of relapse or compared with patients without B-cell recovery.
  • In conclusion these data support the potential of a long-term plateau of survival outcomes in patients receiving obe-cel.
  • A simultaneous audio webcast and replay will be accessible on the events section of Autolus’ website.

Iterum Therapeutics Receives FDA Acceptance of Resubmission of NDA for Oral Sulopenem for the treatment of Uncomplicated Urinary Tract Infections

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пятница, мая 31, 2024
New Drug Application, Patient, Food, NDA, Clinical trial, FDA, Therapy, PDUFA, ITRM, Infection, Woman, Urinary tract infection, Physician, Pharmaceutical industry

Under the Prescription Drug User Fee Act (“PDUFA”), the FDA has deemed the Company’s NDA resubmission to be a Class II complete response which has a six-month review period from the date of resubmission.

Key Points: 
  • Under the Prescription Drug User Fee Act (“PDUFA”), the FDA has deemed the Company’s NDA resubmission to be a Class II complete response which has a six-month review period from the date of resubmission.
  • As a result, the FDA has assigned a PDUFA action date of October 25, 2024.
  • “We are very pleased that the FDA has accepted the resubmission of the NDA for oral sulopenem,” said Corey Fishman, Chief Executive Officer.
  • “This significant milestone brings us one step closer to the potential approval of oral sulopenem for uUTIs providing patients and physicians with a much-needed treatment option for this underserved market.

Additional Data Showing Acoramidis Increases Serum Transthyretin Which is Associated with Improved Cardiovascular Outcomes Presented at ISA from BridgeBio Pharma’s Phase 3 ATTRibute-CM Study in Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

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среда, мая 29, 2024
ACT, Patient, Prognosis, Survival, Amyloidosis, American Heart Association, Month, Risk, Food, ACC, NDA, European Medicines Agency, Calendar, University, ISA, Safety, Serum, ESC, PDUFA, AHA, Congress, Death, Cancer, Biomarker, Cox, Columbia University, Mortality, CVH, MAA, Vancouver General Hospital, American College, Webcast, CVM, Quality of life, TTR, Pharmaceutical industry

ATTRibute-CM was designed to study the efficacy and safety of acoramidis, an investigational, next-generation, orally administered, highly potent, small molecule stabilizer of TTR.

Key Points: 
  • ATTRibute-CM was designed to study the efficacy and safety of acoramidis, an investigational, next-generation, orally administered, highly potent, small molecule stabilizer of TTR.
  • BridgeBio will host an investor call on Wednesday, May 29th at 5:30 pm ET to discuss these results.
  • “The data presented at ISA confirm that improved stabilization as reflected in higher serum TTR levels is directly correlated with improved clinical outcomes.
  • Prior analyses from ATTRibute-CM demonstrated that the near-complete stabilization by acoramidis rapidly and durably increased serum TTR levels.

Milestone Pharmaceuticals Announces FDA Acceptance of New Drug Application for CARDAMYST™

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среда, мая 29, 2024
Patient, Self-management, Paroxysmal supraventricular tachycardia, NDA, Milestone, FDA, PDUFA, Caregiver, PSVT, Pharmaceutical industry

The FDA Prescription Drug User Fee Act (PDUFA) target date is 10 months from the acceptance date of May 26, 2024.

Key Points: 
  • The FDA Prescription Drug User Fee Act (PDUFA) target date is 10 months from the acceptance date of May 26, 2024.
  • “The FDA’s acceptance of our NDA for CARDAMYST brings Milestone one step closer in our mission in providing a new, convenient and effective treatment option for patients with PSVT,” said Joseph Oliveto, President, and Chief Executive Officer of Milestone Pharmaceuticals.
  • “We understand the frequent impact that PSVT has on patients, as well as the underappreciated burden it places on their families and caregivers.
  • Milestone continues to advance commercial preparations to support the anticipated launch of etripamil with the proposed trade name, CARDAMYST.

FDA Grants Priority Review to Merck’s Application for KEYTRUDA® (pembrolizumab) Plus Chemotherapy as First-Line Treatment of Patients With Unresectable Advanced or Metastatic Malignant Pleural Mesothelioma

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среда, мая 29, 2024
Oncology, FDA, Health, Clinical Trials, Pharmaceutical, Biotechnology, MSD, Patient, Progression-free survival, Risk, Food, PFS, PDUFA, Death, ASCO, Society, MRK, Annual general meeting, Pharmaceutical industry

The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date of September 25, 2024.

Key Points: 
  • The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date of September 25, 2024.
  • At 12 months, the estimated PFS rate was 26% for KEYTRUDA plus chemotherapy versus 17% for chemotherapy alone.
  • The ORR was significantly higher for KEYTRUDA plus chemotherapy versus chemotherapy alone (62% versus 38%, p
  • The safety profile of KEYTRUDA plus chemotherapy in this study was consistent with previously reported studies.

SHAREHOLDER ALERT: Pomerantz Law Firm Investigates Claims on Behalf of Investors of Abeona Therapeutics Inc. - ABEO

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вторник, мая 28, 2024
Patient, Symantec Endpoint Protection, News, Food, Concha bullosa, FDA, PDUFA, RDEB, CMC, Pomerantz, LLP, CRL, BLA, Food and Drug Administration

NEW YORK, May 28, 2024 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Abeona Therapeutics Inc. (“Abeona” or the “Company”) (NASDAQ: ABEO).

Key Points: 
  • NEW YORK, May 28, 2024 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Abeona Therapeutics Inc. (“Abeona” or the “Company”) (NASDAQ: ABEO).
  • The investigation concerns whether Abeona and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices.
  • Founded by the late Abraham L. Pomerantz, known as the dean of the class action bar, Pomerantz pioneered the field of securities class actions.
  • The Firm has recovered billions of dollars in damages awards on behalf of class members.

Royalty Pharma and Agios Pharmaceuticals Enter Into Vorasidenib Royalty Agreement for $905 Million

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вторник, мая 28, 2024
Breakthrough therapy, Patient, Progression-free survival, Glioma, Food, Prevalence, FDA, EDT, Trial of the century, PDUFA, IDH, Incidence, Webcast, Pivotal, Food and Drug Administration, INDIGO, Enzyme, Pharmaceutical industry

“We are excited to acquire royalties on vorasidenib, which if approved, would be the first targeted therapy for patients with IDH-mutant glioma,” said Pablo Legorreta, founder and Chief Executive Officer of Royalty Pharma.

Key Points: 
  • “We are excited to acquire royalties on vorasidenib, which if approved, would be the first targeted therapy for patients with IDH-mutant glioma,” said Pablo Legorreta, founder and Chief Executive Officer of Royalty Pharma.
  • Under the terms of the agreement, Royalty Pharma will pay Agios $905 million in upfront cash on FDA approval of vorasidenib in exchange for a 15% royalty on annual U.S. net sales of vorasidenib up to $1 billion and a 12% royalty on annual U.S. net sales greater than $1 billion.
  • Royalty Pharma projects greater than $1 billion in peak annual sales potential for vorasidenib, which is expected to generate royalties of greater than $150 million annually to Royalty Pharma.
  • Royalty Pharma will host a conference call and simultaneous webcast to discuss the transaction today, Tuesday, May 28th at 8:30 a.m. Eastern Time.

Agios Announces $905 Million Purchase Agreement for Vorasidenib Royalty

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вторник, мая 28, 2024
Agios Pharmaceuticals, Food, Goldman Sachs, Sickle cell disease, FDA, Sale, PDUFA, IDH, AGIO, Thalassemia, Glioma, Enzyme, Pharmaceutical industry

CAMBRIDGE, Mass., May 28, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and PK activation pioneering therapies for rare diseases, announced that the company has agreed to sell its rights to its 15% royalty on potential U.S. net sales of Servier’s vorasidenib to Royalty Pharma. Under the terms of the agreement, Agios will receive an upfront payment of $905 million upon approval of vorasidenib by the U.S. Food and Drug Administration (FDA) and Royalty Pharma will receive the entirety of the 15% royalty on annual U.S. net sales of vorasidenib up to $1 billion, and a 12% royalty on annual U.S. net sales greater than $1 billion. Agios will retain a 3% royalty on annual U.S. net sales greater than $1 billion.

Key Points: 
  • – Royalty Pharma to Acquire Rights to Agios’ 15% Royalty on Potential Vorasidenib U.S. Net Sales for $905 Million Upfront upon FDA Approval of Vorasidenib; Agios to Share in Economics Above Certain Revenue Thresholds –
    – In Total, Agios to Receive $1.1 Billion in Payments upon FDA Approval of Vorasidenib; PDUFA Action Date of August 20, 2024 –
    CAMBRIDGE, Mass., May 28, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and PK activation pioneering therapies for rare diseases, announced that the company has agreed to sell its rights to its 15% royalty on potential U.S. net sales of Servier’s vorasidenib to Royalty Pharma.
  • Under the terms of the agreement, Agios will receive an upfront payment of $905 million upon approval of vorasidenib by the U.S. Food and Drug Administration (FDA) and Royalty Pharma will receive the entirety of the 15% royalty on annual U.S. net sales of vorasidenib up to $1 billion, and a 12% royalty on annual U.S. net sales greater than $1 billion.
  • Agios will retain a 3% royalty on annual U.S. net sales greater than $1 billion.
  • As part of that divestiture, Agios is owed a milestone payment of $200 million upon vorasidenib’s approval by the FDA, as well as a 15% royalty on U.S. net sales of vorasidenib.

SHAREHOLDER ALERT: Pomerantz Law Firm Investigates Claims on Behalf of Investors of Abeona Therapeutics Inc. - ABEO

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вторник, мая 21, 2024
Patient, Symantec Endpoint Protection, News, Food, Concha bullosa, FDA, Prior, PDUFA, RDEB, CMC, Pomerantz, LLP, CRL, BLA, Food and Drug Administration

NEW YORK, May 21, 2024 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Abeona Therapeutics Inc. (“Abeona” or the “Company”) (NASDAQ: ABEO).

Key Points: 
  • NEW YORK, May 21, 2024 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Abeona Therapeutics Inc. (“Abeona” or the “Company”) (NASDAQ: ABEO).
  • The investigation concerns whether Abeona and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices.
  • Founded by the late Abraham L. Pomerantz, known as the dean of the class action bar, Pomerantz pioneered the field of securities class actions.
  • The Firm has recovered billions of dollars in damages awards on behalf of class members.

Autolus Therapeutics Reports First Quarter 2024 Financial Results and Business Updates

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пятница, мая 17, 2024
Conference call, UCL, Cardinal Health, Marketing, European Medicines Agency, CD19, FDA, EHA, Multiple myeloma, GD2, Immunotherapy, Research, Flow cytometry, Information technology, University College London, Cardinal, SLE, Conference, BioNTech, MIA, ACS Chemical Biology, MAA, ASH, Clinical trial, Bone marrow, Trial of the century, Nucleus, ADS, ASCO, SME, Minocycline, Marketing Authorisation Application, BCMA, Society, European Hematology Association, Canadian Aviation Regulations, Patient, Nature Communications, T cell, Quarter, Therapy, Engineering, Autoimmune disease, PDUFA, ALL, EMA, TRBC, Abstract, BLA, GMP

Two patients have been enrolled and Autolus continues to expect initial clinical data in late 2024.

Key Points: 
  • Two patients have been enrolled and Autolus continues to expect initial clinical data in late 2024.
  • Enrollment of the initial cohorts are complete and further updates from the MCARTY study are anticipated in H2 2024.
  • In April 2024, Autolus announced the appointment of Mike Bonney as Chairman of the Board, and Ravi Rao M.D., as Non-Executive Director.
  • Financial Results for the Quarter Ended March 31, 2024