Published in Pipeline

Aldeyra submits NDA for primary vitreoretinal lymphoma treatment

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Aldeyra Therapeutics announced the submission of a new drug application (NDA) to the FDA for ADX-2191 (intravitreal methotrexate 0.8%) for the treatment of primary vitreoretinal lymphoma (PVRL).

Didn’t Aldyera just submit an NDA?

They did! Last month the company submitted an NDA for 0.25% topical ocular reproxalap to treat dry eye disease (DED). Read about it here.

Tell me about ADX-2191.

The company’s investigational drug candidate is an intravitreally-administered, non-compounded formulation of methotrexate. The FDA previously granted ADX-2191 Orphan Drug and Fast Track designation for the treatment of retinitis pigmentosa, proliferative vitreoretinopathy (PVR), and PVRL.

What makes it unique?

ADX-2191 is the first sterile, non-compounded formulation of methotrexate designed for intravitreal administration for specific rare retinal diseases.

How did it perform in clinical trials?

The phase 3 GUARD trial assessed the use of ADX-2191 compared to standard-of-care for the prevention of proliferative vitreoretinopathy (PVR) in patients for 6 months. Recurrence rates of ADX-2191 over 6 weeks were found to be well tolerated with no noted safety signals. (via)

Any adverse events?

While there were no observed treatment-emergent serious adverse events (AEs), the most common reported AE was mild punctate keratitis (16%)—a frequent side effect of intravitreal methotrexate.

What’s next?

A Typic C meeting with the FDA for ADX-2191 for PVR is planned for the first half of 2023, as well as phase 2 trial results on ADX-2191 for RP.

Due to its Orphan Drug designation, Aldeyra has requested Priority Review designation for ADX-2191. This shorten’s the FDA’s standard 10-month review period for potential market approval to 6 months.

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