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Graphite Bio Announces U.S. FDA Fast Track Designation Granted to GPH101 for the Treatment of Sickle Cell Disease

May 25, 2022

by administrator BPG Blog

“The FDA’s decision to grant Fast Track Designation to GPH101 for sickle cell disease signifies the need for novel medicines for this serious genetic disease and supports the ongoing development of our unique gene correction approach that we believe could offer a definitive cure for sickle cell patients,” said Josh Lehrer, M.D., M.Phil., chief executive officer of Graphite Bio. “This designation has the potential to accelerate the development of GPH101, which we are advancing with the goal of precisely and efficiently correcting the genetic mutation that is the underlying cause of sickle cell disease. We continue to enroll patients in our Phase 1/2 CEDAR trial and expect to dose our first patient later this year, with initial proof-of-concept data anticipated next year.”

The FDA’s Fast Track program facilitates the expedited development and review of new drugs or biologics that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs. GPH101 was previously granted orphan drug designation by the FDA.

About GPH101 for Sickle Cell Disease
GPH101 is an investigational next-generation gene-edited autologous hematopoietic stem cell (HSC) therapy designed to directly correct the genetic mutation that causes sickle cell disease (SCD). SCD is a serious, life-threatening inherited blood disorder that affects approximately 100,000 people in the United States and millions of people around the world, making it the most prevalent monogenic disease worldwide. GPH101 is the first investigational therapy to use a highly differentiated gene correction approach that seeks to efficiently and precisely correct the mutation in the beta-globin gene to decrease sickle hemoglobin (HbS) production and restore adult hemoglobin (HbA) expression, thereby potentially curing SCD.

Click here to read the full article at BusinessWire

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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.

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

May 6, 2022

by administrator BPG Blog

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

aTyr Pharma Announces FDA Orphan Drug Designation for Efzofitimod (ATYR1923) for Treatment of Systemic Sclerosis

April 18, 2022

by administrator BPG Blog

aTyr Pharma, Inc. (Nasdaq: LIFE), a clinical stage biotherapeutics company engaged in the discovery and development of innovative medicines based on novel biological pathways, today announced that the U.S. Food and Drug Administration (FDA) has granted the company orphan drug designation for its lead therapeutic candidate, efzofitimod, for the treatment of systemic sclerosis (SSc, also known as scleroderma).

Efzofitimod is a potential first-in-class immunomodulator that downregulates innate and adaptive immune responses in uncontrolled inflammatory disease states via selective modulation of neuropilin-2 (NRP2). Clinical proof-of-concept was recently established for efzofitimod in a Phase 1b/2a study in patients with pulmonary sarcoidosis, a major form of interstitial lung disease (ILD). Many patients with SSc may develop associated ILD, known as SSc-ILD. The pathology of SSc-ILD is driven by the same immune cells that are central to sarcoidosis pathology, and NRP2 is upregulated on these cells, particularly on macrophages. Furthermore, efzofitimod has been shown to reduce lung and skin fibrosis in animal models of SSc and idiopathic pulmonary fibrosis, where it matched or outperformed known anti-fibrotic agents, including nintedanib and pirfenidone.

“We are very pleased to receive orphan drug designation for efzofitimod for SSc, which marks the second such designation for our efzofitimod clinical program,” said Sanjay S. Shukla, M.D., M.S., President and CEO of aTyr. “The data we have presented in animal models of SSc along with the positive findings reported from our recent Phase 1b/2a study in pulmonary sarcoidosis patients suggest that efzofitimod has the potential to be a new treatment option that resolves inflammation and subsequent fibrosis in those living with SSc-ILD. We look forward to exploring the potential expansion of our efzofitimod clinical program into other forms of ILD with high unmet need where this novel therapeutic may be able to improve patient outcomes.”

Click here to read the full article at GlobeNewswire

Gilead’s Kite flies higher as Yescarta leads CAR-T therapy class to earlier lymphoma treatment

April 13, 2022

by administrator BPG Blog

Kite Pharma CEO Christi Shaw has put the eligible U.S. patient population for Yescarta in the second-line setting at 14,000 compared with 8,000 patients for its original third-line use. The new indication could get Yescarta $1.5 billion in peak sales over time, according to analysts at RBC Capital Markets.

The optimism—and the FDA nod—came from the phase 3 Zuma-7 trial. The study showed Yescarta reduced the risk of disease progression, death or the need for a new therapy by 60.2% compared to standard of care, which involves chemotherapy and stem cell transplant. At two years, 40.5% of Yescarta takers were still alive and didn’t require additional cancer treatment or experience cancer progression, versus 16.3% for the control arm.

It’s also worth noting that, while 94% of patients in the Yescarta group successfully received the CAR-T therapy, only 36% of those in the control arm actually made it to stem cell transplant, Shaw noted during an interview.

Click here to read the full article at Fierce Pharma