FDA pathway · 505(b)(1) · Orphan + priority review voucher

Dose precision is the product.

Children with rare diseases are often dosed from adult tablets cut at home. One validated formulation prints every weight band at ±1%.

October 2026Orphan + rare pediatric designation applications

Q1 2027Pre-IND meeting with the FDA

2027–28Clinical program

Target Q4 2028NDA submission

From a quartered adult tablet to a printed dose for each weight band.

Today

One adult strength, quartered at home

Dose accuracy: unknown

Every new strength is a separate campaign no manufacturer tools up for

With PharmaTher

Printed, weight-banded, taste-masked chewables

A new strength, or a titration series as the child grows, is a file change, not a factory change.

Lead FDA-track programNDA target Q4 2028

Undisclosed compound · rare pediatric disease

Serious, early-onset and dosed by body weight: a dose that must change as the child grows

No FDA-approved product exists; today's treatment is improvised

Printed weight-banded, taste-masked chewable in place of hand-measured doses

Designation applications draw on published literature; an objective endpoint supports a compact, single-arm program

PharmaTher will announce the designation applications once filed. Further candidates are screened behind it.

Next: ultra-rare diseases

Too few patients for a production line. Exactly right for printing.

Ultra-rare diseases affect fewer than 1 in 50,000 people, or roughly 7,000 or fewer in the U.S. These patients may need only a few thousand doses a year, often in weight-based strengths.

PharmaTher is evaluating ultra-rare diseases with no FDA-approved treatment where a printed, precisely dosed product could become the first approved therapy, using the same regulatory approach as its lead program.

~10,000Known rare diseases

~95%Have no FDA-approved treatment

FDA Rare Disease Evidence Principles (2025)For certain genetic diseases affecting fewer than 1,000 U.S. patients, approval can rest on one adequate and well-controlled study plus confirmatory evidence.

Sources: U.S. National Institutes of Health (NCATS); U.S. FDA, Rare Disease Evidence Principles (September 2025).

The voucher opportunity

Approval can earn a priority review voucher, a potential US$100M+ asset.

Orphan drug designation7 years of market exclusivity, an NDA fee waiver and a clinical-trial tax credit.

Rare pediatric disease designationA designated, never-approved active approved under a full NDA earns a voucher.

The priority review voucherA transferable ticket to a six-month FDA review of any future drug. Large pharma buys them; small companies sell them.

What rare pediatric vouchers have sold for (US$M, reported)

$350

$125

$110

$150

$155

US$100–150MCurrent market value of a voucher

US$513MPaid for vouchers in 2024, the busiest year on record

Compound and indication undisclosed. Timelines and program costs are internal estimates subject to FDA feedback. Voucher award and value are not assured; eligibility is determined at approval. Sources: 21 USC 360ff; FDA OOPD; BioSpace, "Priority Review Vouchers: By the Numbers" (Mar 2025); company press releases on each sale.

Partner on rare pediatric programs

Co-develop, license or fund a program with PharmaTher.

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Investors

The corporate presentation covers the program, the voucher and every catalyst.

Download the deck (PDF)