BPG BLOG (31)

Allogene Therapeutics Receives FDA Orphan-Drug Designation for ALLO-605, its First TurboCAR™ T Cell Product Candidate, for the Treatment of Multiple Myeloma

SOUTH SAN FRANCISCO, Calif., April 27, 2022 (GLOBE NEWSWIRE) — Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T™) products for cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation (ODD) to ALLO-605, the Company’s next-generation AlloCAR T product candidate targeting BCMA for the treatment of multiple myeloma.

ALLO-605 is the Company’s first TurboCAR™ product candidate. TurboCAR™ is a proprietary, next generation platform technology based on a programmable cytokine signaling, designed to control T cell exhaustion and to improve T cell function and potency. These properties may enable CAR T products to succeed in more difficult to treat hematologic malignancies and solid tumors. The FDA granted Fast Track designation to ALLO-605 in Q2 2021 based on the potential for the product candidate to address an unmet need for patients who have failed other standard multiple myeloma therapies. The Phase 1 study evaluating ALLO-605 is ongoing.

“Orphan-drug designation marks an important step towards developing our anti-BCMA portfolio for patients with multiple myeloma and making allogeneic CAR T products readily available for patients,” said Rafael Amado, M.D., Executive Vice President of Research and Development and Chief Medical Officer. “We look forward to providing an update on our BCMA clinical assets by the end of the year with an eye toward prioritizing a strategy for the next stage of development.”

Click here to read the full article at GlobeNewswire

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

BPG BLOG (30)

ENHERTU® (fam-trastuzumab deruxtecan-nxki) granted Breakthrough Therapy Designation in the US for patients with HER2-low metastatic breast cancer

WILMINGTON, Del.–(BUSINESS WIRE)–AstraZeneca and Daiichi Sankyo’s ENHERTU® (fam-trastuzumab deruxtecan-nxki)has been granted Breakthrough Therapy Designation (BTD) in the US for the treatment of adult patients with unresectable or metastatic HER2-low (IHC 1+ or IHC 2+/ISH-negative) breast cancer who have received a prior systemic therapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy. Patients with hormone receptor (HR) positive breast cancer should additionally have received or be ineligible for endocrine therapy.

ENHERTUis a specifically engineered HER2-directed antibody drug conjugate (ADC) being jointly developed and commercialized by AstraZeneca and Daiichi Sankyo.

The Food and Drug Administration’s (FDA) BTD is designed to accelerate the development and regulatory review of potential new medicines that are intended to treat a serious condition and address a significant unmet medical need. The new medicine needs to have shown encouraging preliminary clinical results that demonstrate substantial improvement on a clinically significant endpoint over available medicines.

Up to half of all patients with breast cancer have tumors with a HER2 immunohistochemistry (IHC) score of 1+, or 2+ in combination with a negative in-situ hybridization (ISH) test, a level of HER2 expression not currently eligible for HER2-targeted therapy.1-4 Low HER2 expression occurs in both HR-positive and HR-negative disease.5

Click here to read the full article at BusinessWire

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

5.10

Editas sees clinical promise with new FDA pediatric disease tag for another blood disorder

Following a management shake-up, Editas Medicine received some welcome good news. The biotech has scored a second rare pediatric disease designation from the FDA for its gene edited medicine EDIT-301.

EDIT-301 already holds the designation for treating sickle cell disease and is currently under investigation in a clinical study. Following the FDA’s most recent designation, Editas expects to launch a phase 1/2 study of EDIT-301 in patients with transfusion-dependent beta thalassemia this year.

The gene editing biotech has been making headlines recently, but not necessarily for its clinical treatments. The company announced several leadership shake-ups, bringing on Sarepta’s Chief Medical Officer Gilmore O’Neill to replace Chairman, President and CEO James Mullen, who will become Editas’ executive chairman, effective June 1. Interestingly, O’Neill himself isn’t filling the biotech’s chief medical officer role, which currently sits empty after the company ousted Lisa Michaels, M.D., without explanation in February.

Click here to read the full article at Fierce Biotech

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

BPG BLOG (27)

Ocugen Sees Ray of Light with Gene Therapy for Retinitis Pigmentosa

Ocugen, which has faced a string of delays with its Bharat Biotech-partnered vaccine, reported some good news today.

The company, based in Malvern, Pennsylvania, reported that the independent Data and Safety Monitoring Board (DSMB) involved in the Phase I/II trial of OCU400, a gene therapy in development for Retinitis Pigmentosa (RP), recommended the study proceed with enrolling more patients. The trial is evaluating the modifier gene therapy OCU400 for RP caused by mutations in the nuclear receptor subfamily 2 group E member 3 (NR2E3) and Rhodopsin (RHO) genes. Ocugen recently dosed the first patient in the study. The DSMB recommended the company continue enrolling the remaining subjects in the current cohort at the target dose level.

RP is a family of rare eye diseases affecting the retina. RP results in the retina breaking down over time, causing vision loss. Symptoms typically begin in childhood, but the majority of patients eventually lose most of their vision. The most common early symptom is loss of night vision.

Click here to read the full article at BioSpace

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

BPG BLOG (26)

After 18 years in development, AstraZeneca’s CTLA-4 antibody gets the red carpet treatment at the FDA

After a decade, a second CTLA-4 antibody may finally be on its way to approval.

AstraZeneca announced Monday that the FDA had given priority review to tremelimumab, its long-gestating CTLA-4 antibody, in combination with the PD-L1 inhibitor Imfinzi for patients with liver cancer that can’t be treated with surgery.

The Big Pharma’s application is based on a multi-year, 1,324-patient study that showed 30.7% of patients who received a “priming dose” of tremelimumab followed by Imfinzi were alive after three years, compared to 20.2% of patients who received Bayer’s sorafenib, the standard of care.

Susan Galbraith, AstraZeneca’s cancer R&D chief, called that data “unprecedented.” Others raised questions about how the data held up compared to an alternative treatment regimen being developed by Roche, and how much benefit the CTLA-4 antibody offered, given that patients who received Imfinzi alone showed nearly identical survival at the two-year mark. The difference only emerged after three years.

Click here to read the full article at Endpoints News

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

BPG BLOG (23)

Quanterix’s blood test for multiple sclerosis relapse risk nabs FDA breakthrough nod

Though relapsing-remitting multiple sclerosis, or RRMS, can be managed with many of the approved treatments for MS symptoms, there is still not scientifically proven, FDA-cleared way to predict when or if another relapse period will occur.

There may be one on the horizon, however, since Quanterix recently received the regulator’s breakthrough device designation for a blood test designed to do exactly that.

In RRMS, the most common form of multiple sclerosis, periods of stable remission are interrupted by relapses of new or worsening symptoms—like visual loss or double vision, numbness, weakness and balance issues—that last at least 48 hours. Quanterix’s test is designed to look for signs that a patient will relapse within the next four years.

Click here to read the full article at Fierce Biotech

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

BPG BLOG (22)

CHMP adopts positive opinion for Filsuvez® for the treatment of Dystrophic and Junctional EB

DUBLIN, Ireland, and Boston MA, April 22, 2022, Amryt (Nasdaq: AMYT), a global, commercial-stage biopharmaceutical company focussed on acquiring, developing and commercializing novel treatments for rare diseases, is pleased to announce that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending the approval of Filsuvez® in the European Union (EU) for the treatment of partial thickness wounds associated with dystrophic and junctional Epidermolysis Bullosa (EB) in patients 6 months and older.  EB is a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment.

Based on this CHMP recommendation a decision by the European Commission (EC) is expected on the Filsuvez® application within 67 days.  The centralised marketing authorisation would be valid in all EU Member States as well as in Iceland, Liechtenstein, and Norway.  The Medicines and Healthcare products Regulatory Agency (MHRA) in the UK is expected to grant authorisation within the same time period.

The CHMP positive opinion is supported by Phase 3 data from the EASE trial which was the largest ever global trial conducted in patients with EB, performed across 58 sites in 28 countries.

Click here to read the full article at AMRYT Pharma

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

BPG BLOG (21)

Nkarta’s NK Cell Therapies for Blood Cancer Tout Full Recoveries

Nkarta, a biopharmaceutical company that engineers natural killer cell therapies to treat cancer, announced that two of its therapies have shown complete remission in patients with blood cancer. The exciting results from the Phase I studies are some of the latest hopeful developments in the biopharma space for difficult-to-treat blood cancers.

San Francisco-based Nkarta develops therapies in the oncology space, including for blood cancers. The company has two leading candidates, drugs NKX101 and NKX019.

In a Phase I clinical trial, Nkarta tested NKX101 on patients with relapsed or refractory acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). Because of the difficulty of treating AML, there is currently no standard of care. Nkarta received Orphan Drug Designation from the U.S. Food and Drug Administration for NKX101 in December 2021.

“Relapsed/refractory acute myeloid leukemia (AML) is a historically hard-to-treat disease, and given the lack of effective treatments, people with cancer and those who treat them are faced with few options,” Marcello Rotta, M.D., who works at the Colorado Blood Cancer Institute and was an investigator in the NKX101 clinical trial, said in a statement.

Click here to read the full article at BioSpace

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

BPG BLOG (19)

Zambon Receives U.S. FDA Breakthrough Therapy Designation for CMS I-neb® in Patients with Non-Cystic Fibrosis Bronchiectasis (NCFB)

MILAN and BOSTON, April 21, 2022 /PRNewswire/ — Zambon, a multinational pharmaceutical company focused on innovating cure and care to improve people’s health and the quality of patients’ lives, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to colistimethate sodium powder for nebulization solution (CMS I–neb®) for the reduction in the incidence of pulmonary exacerbations in adult patients with non-cystic fibrosis bronchiectasis (NCFB) colonized with P. aeruginosa. NCFB is a chronic, progressive, and irreversible respiratory disease. There are no approved inhaled treatments currently available for patients with bronchiectasis and chronic P. aeruginosa colonization.

The Breakthrough Therapy Designation is supported by data from the Phase 3 PROMIS – I study, which showed that CMS I-neb® significantly reduced the annual rate of exacerbations in patients with NCFB and P. aeruginosa chronic infection, the primary endpoint of the trial. In addition, the trial met important secondary endpoints, including reduction of severe exacerbations and prolongation of time to first exacerbation compared to placebo, and also improvement in Quality of Life (QoL). The treatment was demonstrated to be well tolerated with adverse events similar between groups. Data from the Phase 3 trial were most recently presented at the European Respiratory Society (ERS) International Congress in September 2021.

With no approved drugs for patients with NCFB colonized by P. aeruginosa anywhere in the world, the Breakthrough Therapy Designation by FDA marks an important step forward in support of our mission to develop and provide treatment options for people with rare and severe respiratory diseases,” said Roberto Tascione, CEO at Zambon. “We are proud that the FDA has recognized the importance of CMS I–neb® and the urgent need to develop innovative treatments for these patients.”

Click here to read the full article at Cision PR Newswire

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

BPG BLOG (17)

FDA Grants Regenerative Medicine Advanced Therapy (RMAT) Designation to AlloVir’s Posoleucel for Prevention of Multiple Life-Threatening Infections from Six Viruses in Allogeneic Hematopoietic Cell Transplant Patients

AlloVir (Nasdaq: ALVR), a late clinical-stage allogeneic T cell immunotherapy company, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its lead investigational multi-virus-specific T cell therapy, posoleucel, for the prevention of clinically significant infections and disease from six devastating viruses that commonly impact high-risk adult and pediatric patients following allogeneic hematopoietic cell transplant (allo-HCT) – adenovirus (AdV), BK virus (BKV), cytomegalovirus (CMV), Epstein-Barr virus (EBV), human herpes virus-6 (HHV-6) and JC virus (JCV). This is the third RMAT designation that FDA has granted to posoleucel, in recognition of the therapy’s transformative potential to address significant unmet medical needs facing immunocompromised allo-HCT patients.

The FDA previously granted RMAT designation to posoleucel for the treatment of hemorrhagic cystitis (HC) caused by BKV in adults and children following allo-HCT and for the treatment of adenovirus infection following allo-HCT. RMAT designation enables early interactions with the FDA to discuss clinical trial design and other actions to expedite development and review. Outside of the United States, the European Medicines Agency has granted posoleucel PRIority Medicines (PRIME) designation for the treatment of serious infections with AdV, BKV, CMV, EBV and HHV-6.

“The receipt of three RMAT designations for a single therapy is unprecedented. Posoleucel’s three RMAT designations reflect the strength of AlloVir’s multi-virus platform and its potential both to deliver an important treatment option for immunocompromised patients who currently have none, and to transform the management of allo-HCT patients with a multi-virus prevention approach,” said Ercem Atillasoy, M.D., Chief Regulatory and Safety Officer, AlloVir.

Click here to read the full article at Business Wire

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.