Is kebilidi the same as upstaza?

The U.S. Food and Drug Administration (FDA) approved Kebilidi in November 2024, approximately two years after the same gene therapy was approved in the European Union (EU) as Upstaza. This means that when asking, 'Is kebilidi the same as upstaza?', the answer is yes: they are different brand names for the exact same medical product, eladocagene exuparvovec.

Quick Summary

Kebilidi and Upstaza are two different brand names for the single gene therapy, eladocagene exuparvovec, developed by PTC Therapeutics to treat AADC deficiency. Kebilidi is used in the U.S., while Upstaza is used in the EU and U.K. The distinction is based purely on regional branding and regulatory approval.

Key Points

• Shared Identity: Kebilidi and Upstaza are two brand names for the exact same gene therapy, eladocagene exuparvovec.

• Regional Branding: The name difference is due to geographic regulatory approval; Kebilidi is used in the U.S. while Upstaza is used in the E.U. and U.K..

• AADC Deficiency Treatment: This therapy is designed to treat AADC deficiency, a rare genetic disorder affecting neurotransmitter production and motor function.

• Gene Replacement: The treatment works by delivering a functional DDC gene via a harmless virus vector into the brain's putamen.

• Surgical Delivery: The gene therapy is administered as a one-time intraputaminal infusion via a minimally invasive stereotactic neurosurgical procedure.

• Improved Outcomes: Clinical trials showed that treated patients achieved significant motor milestones that are typically not seen in untreated individuals with severe AADC deficiency.

• Known Manufacturer: Both products are manufactured by the global biopharmaceutical company PTC Therapeutics.

Understanding the Shared Generic Name: Eladocagene Exuparvovec

Kebilidi and Upstaza are identical, sharing the generic name eladocagene exuparvovec. This one-time gene therapy from PTC Therapeutics treats Aromatic L-amino acid decarboxylase (AADC) deficiency by addressing its genetic cause. The different brand names are a result of separate regulatory approvals and marketing choices in different regions. Upstaza received approval in Europe first, followed by Kebilidi's approval in the United States.

Why Different Brand Names Exist

It's common for a single drug to have multiple brand names due to regional branding, regulatory factors, or acquisitions. PTC Therapeutics secured marketing authorization in various markets at different times, opting for unique names for each region.

The Condition Treated: AADC Deficiency

Eladocagene exuparvovec treats AADC deficiency, a rare genetic disorder caused by mutations in the DDC gene. This leads to a lack of the AADC enzyme needed to produce vital neurotransmitters like dopamine and serotonin. The resulting impaired nervous system function causes severe symptoms, especially in children, including delayed motor skills, weak muscle tone, movement disorders, and developmental delays. Before this gene therapy, treatments focused only on managing symptoms.

Symptoms of AADC deficiency can include:

• Delayed or absent gross motor function
• Weak muscle tone (hypotonia)
• Movement disorders (dyskinesia, oculogyric crises)
• Autonomic dysfunction
• Developmental and cognitive delays

How Eladocagene Exuparvovec Works

The therapy introduces a functional DDC gene copy into the brain's putamen using a harmless adeno-associated virus (AAV) vector. This allows cells to produce the missing AADC enzyme and synthesize neurotransmitters. The treatment is administered through a stereotactic neurosurgical procedure.

Key steps in the treatment process include:

• Pre-operative brain imaging
• Image-guided gene therapy delivery
• Post-operative monitoring

Comparison Table: Kebilidi vs. Upstaza

Feature	Kebilidi	Upstaza
Generic Name	Eladocagene Exuparvovec	Eladocagene Exuparvovec
Manufacturer	PTC Therapeutics	PTC Therapeutics
Regulatory Body	U.S. Food and Drug Administration (FDA)	European Medicines Agency (EMA)
Primary Region	United States	European Union (EU) and United Kingdom (U.K.)
Approval Date	November 2024	July 2022
Target Population	Pediatric and adult patients across the full spectrum of disease severity	Pediatric patients 18 months and older with a severe phenotype

Efficacy and Safety Profile

Clinical trials showed that eladocagene exuparvovec provided significant, long-term benefits, with many patients achieving motor milestones previously unattainable. The FDA noted it offers hope for patients with this devastating condition.

Potential side effects include:

• Dyskinesia
• Fever
• Hypotension
• Anemia
• Increased saliva production
• Insomnia
• Low electrolyte levels

Neurosurgery also carries risks. Safe administration requires specialized medical teams in designated centers, as recommended by the European Medicines Agency.

Conclusion

Kebilidi and Upstaza are the same gene therapy (eladocagene exuparvovec) by PTC Therapeutics, differing only by brand name due to regional approvals. This one-time therapy treats AADC deficiency by correcting the genetic defect, aiming to restore neurotransmitter production. While not a traditional cure, it has shown significant improvements in motor function and quality of life. Patients should consult their doctor to discuss treatment details, outcomes, and risks.

Frequently Asked Questions

Are Kebilidi and Upstaza exactly the same medication?

Yes, Kebilidi and Upstaza are the exact same gene therapy product, known by the generic name eladocagene exuparvovec. The different brand names are used for marketing and regulatory purposes in different parts of the world.

Why are there different names for the same therapy?

The names differ because of different regulatory approvals in different regions. PTC Therapeutics, the manufacturer, received European marketing authorization first for Upstaza and later obtained FDA approval in the United States for Kebilidi.

What is the generic name for Kebilidi and Upstaza?

The generic name for both brand names is eladocagene exuparvovec.

What condition does this gene therapy treat?

The therapy is indicated for the treatment of Aromatic L-amino acid decarboxylase (AADC) deficiency, a rare genetic disorder that affects the nervous system.

How is the gene therapy administered?

The therapy is delivered as a one-time infusion directly into a specific region of the brain called the putamen, using a minimally invasive stereotactic neurosurgical procedure.

What are the common side effects of Kebilidi/Upstaza?

Common side effects include dyskinesia (involuntary movements), fever, low blood pressure, anemia, increased saliva production, and insomnia.

Which company manufactures Kebilidi/Upstaza?

Both Kebilidi and Upstaza are manufactured by PTC Therapeutics, a global biopharmaceutical company specializing in rare disorders.

Is this a permanent cure for AADC deficiency?

The long-term effects of the gene therapy are still being monitored. While it addresses the genetic root cause and has shown significant improvement in motor function and developmental milestones, it is considered a life-altering treatment rather than a definitive cure.