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Looking for 2020 FDA decisions: A diverse group of cell, gene and tissue therapies

August 13, 2020
We continue to see lots of activity in the cell, gene and tissue therapy arena: manufacturers who are almost ready to file, those who have recently filed, those who are looking at upcoming PDUFA dates, and those still hoping to get a 2020 approval.

Below are the top therapies that may be approved in 2020.

Brand name/generic name: Instilidrin™ (nadoferagene giradenovec)

Manufacturer: FerGene
Indication: Bladder cancer
Mechanism: Non-traditional gene therapy
Anticipated approval: 2020

FerGene’s Instilidrin is being reviewed by the U.S. Food and Drug Administration (FDA) for treatment of non-muscle invasive bladder cancer (NMIBC).

Bladder cancer is one of the most frequently occurring cancers. Nearly 700,000 people in the U.S. live with bladder cancer. More than 60,000 new cases of NMIBC are diagnosed every year. Close to half of the people with high-grade NMIBC are at an increased risk of recurrence after treatment with Bacillus Calmette-Guérin (BCG).1?

A Phase 3 trial of Instildrin demonstrated a three-month complete response rate of 53% in patients with BCG unresponsive high-grade NMIBC. The single-arm study also demonstrated broad efficacy in this difficult-to-treat patient population with a 73% high growth recurrence-free (HGRF) survival in patients with papillary disease at three months, and 44% HGRF survival at 12 months.2

Brand name/generic name: Roctavian™ (valoctocogene roxaparvovec, BMN 270)

Manufacturer: BioMarin
Indication: Severe hemophilia A
Mechanism of action: Gene therapy
Anticipated approval: August 21, 2020

BioMarin’s Roctavian is being reviewed by the FDA as a one-time gene therapy for adults with hemophilia A.

Hemophilia A is a genetic disease caused by the deficiency of clotting factor VIII. It is the most common type of hemophilia and occurs more frequently in males.3 In the United States, the prevalence of hemophilia A is 20 cases per 100,000 males. Approximately 50-60% of patients with hemophilia A have a severe case (FVIII <2% of normal). This is associated with the severest bleeding episodes.

The Roctavian Phase 3 clinical trial, GENEr8-1, demonstrated an 85% reduction in mean annual bleed rate (ABR) from baseline levels. At this level, all patients were on standard of care prophylaxis prior to Roctavian (9.9 versus 1.5) and 94% reduction in mean FVIII infusions annualized between weeks five to 26 (146.1 versus 6.8).

If approved, BioMarin is considering a price range of $2 million to $3 million for the one-time therapy per treated member.4

Brand name/generic name: Breyanzi™ (JCAR017, lisocabtagene maraleucel, liso-cel)

Manufacturer: Bristol-Myers Squibb Indication: R/R large B cell lymphoma
Mechanism: CAR-T cell therapy
Anticipated approval: November 16, 2020

The FDA is reviewing the BLA for Bristol-Myers Squibb’s Breyanzi (JCAR017, lisocabtagene maraleucel, liso-cel) for the treatment of adult patients with relapsed or refractory (R/R) large B-cell lymphoma, after at least two prior therapies.

Diffuse large B-cell lymphoma (DLBCL) is the most common and aggressive non-Hodgkin’s lymphoma, accounting for three out of every five cases. Approximately one-third of patients with DLBCL relapse after receiving first-line treatment. Historically the median life expectancy for a patient with relapse or refractory is approximately six months.5

TRANSCEND NHL 001, a Phase 1 trial, achieved a complete response rate of 53% (the patients had an absence of all detectable cancer after treatment). The overall patient response rate was 73% (this includes patients who had a partial response as well.5

The FDA has extended its review timeline to November, based on new material BMS submitted after the filing deadline.6

Brand name/generic name: RVT-802

Manufacturer: Enzyvant
Indication: Pediatric congenital athymia
Mechanism of action: Tissue engineering
Anticipated approval: 2020

Enzyvant’s RVT-802 is a one-time tissue-based regenerative therapy for patients with pediatric congenital athymias.

Congenital athymia is a rare and deadly condition associated with complete DiGeorge anomaly (cDGA), CHARGE syndrome and FOXN1 deficiency.7 Children with congenital athymia are born without a thymus, which makes them severely immunodeficient. According to Enzyvant, approximately 20 infants a year are born with congenital athymia in the United States.

Currently there are no FDA-approved therapies for this condition.

The Kaplan-Meier estimates of the patient survival rate — after taking RVT-802 — at year one and year two post treatment were 76% and 75% respectively.7

In December 2019, the FDA issued a Complete Response Letter (CRL) due to concerns about its manufacturing. Enzyvant is anticipated to resubmit RVT-802 for review and hopes to gain approval in 2020.7

Brand name/generic name: Ryoncil™ (remestemcel-L)

Manufacturer: Mesoblast Indication: Graft-versus-host disease
Mechanism of action: Cell therapy
Anticipated approval: September 2020

Mesoblast’s Ryoncil is being reviewed by the FDA for treatment of pediatric steroid-refractory acute graft-versus-host disease. There are approximately 30,000 allogeneic bone marrow transplants globally per year. One quarter of all cases fall within the pediatric population.8

Ryoncil is a donor-derived mesenchymal stem cell (MSC) therapy. It repopulates the bone marrow stroma and supports engraftment of hematopoietic stem cells from the same donor. 8

In Phase 3 clinical trials, the overall response rate was achieved in 70% of patients at day 28. At day 100, 75% of patients were alive compared with 10% to 30% of non-responders, and the survival rates of historical controls.9

Brand name/generic name: PTC-AADC (eladocagene exuparvovec)

Manufacturer: PTC Therapeutics
Indication: Aromatic L-amino acid decarboxylase (AADC) deficiency
Mechanism of action: Gene therapy
Anticipated approval: 2020

PTC Therapeutics will submit a BLA to the FDA for PTC-AADC in the second half of 2020. PTC-AADC is a gene therapy for treatment of patients living with aromatic L-amino acid decarboxylase (AADC) deficiency.10 About 100 people worldwide have this condition.

Three Phase 1 and 2 clinical trials enrolled 26 children from 12 months to 8.5 years. One-time treatment with PTC-AADC improved their Peabody Developmental Motor Scales (PDMS-2), a measure of motor development in children.11


  11. 11. * Anticipated approval dates are predictions made by Prime Therapeutics, based on industry information.

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