FDA Decision Watch: BMY, EGTX Face Key Decisions This Week As BFRI, SRRK And MIRM Score Early Approvals

• Bristol Myers Squibb faces an FDA decision on Sept. 30 for expanding Camzyos to adolescents with obstructive hypertrophic cardiomyopathy.
• Scholar Rock’s Isembyld and Biofrontera's (BFRI) Ameluz received FDA clearance ahead of their scheduled late-September PDUFA dates.
• Mirum Pharmaceuticals’ Atebrioz got FDA approval on Friday for patients aged 12-18 with an ultra-rare genetic disease.

Biotech and pharmaceutical investors have key U.S. Food and Drug Administration (FDA) events to watch this week, including decisions on treatments for a rare genetic disorder and a heart condition, while several other drugs originally scheduled for late-September decisions have already received regulatory clearance.

Bristol Myers Squibb (BMY) awaits an FDA decision on expanding Camzyos to adolescents with obstructive hypertrophic cardiomyopathy, a condition in which thickened heart muscle blocks blood flow. Stockholm-listed Egetis Therapeutics also faces a decision on Emcitate for MCT8 deficiency, a rare genetic disorder that affects how thyroid hormones reach the brain and other tissues.

Meanwhile, Biofrontera (BFRI) and Scholar Rock (SRRK) entered the week with decisions originally scheduled for this period, while Mirum Pharmaceuticals (MIRM) and Incyte (INCY) received FDA approval for Atebrioz late last week.

A PDUFA date is the target deadline by which the FDA aims to complete its review of a drug application.

Bristol Myers Awaits Camzyos Label Expansion

The FDA is scheduled to make a decision on Bristol Myers Squibb (BMY) on September 30. The regulatory body will decide whether to expand the use of Camzyos (Mavacamten) to adolescents ages 12 to under 18 with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).

Obstructive HCM is a condition in which the heart muscle becomes abnormally thick, potentially blocking blood flow out of the heart. Patients can experience shortness of breath, fatigue and chest pain.

Camzyos is already FDA-approved for certain adults with symptomatic oHCM. Bristol Myers said the safety profile among adolescents was similar to that previously observed in adults.

If cleared, Camzyos would become the first cardiac myosin inhibitor approved in the U.S. for adolescents with oHCM, according to the company.

Egetis Awaits FDA Decision On Rare Genetic Disorder Drug

Egetis Therapeutics faces a September 28 decision on Emcitate for MCT8 deficiency.

If approved, Emcitate would become the first FDA-approved treatment for MCT8 deficiency in the U.S. The drug is already approved in the European Union, and the FDA has already granted the application Priority Review. Emcitate has also received Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease designations from the FDA.

FDA Already Acted On Several Late-September Decisions

Several other drugs that had been heading toward FDA decisions around this week have already received regulatory clearance.

Scholar Rock’s (SRRK) Isembyld was scheduled for a September 30 PDUFA date, but the FDA approved the treatment on Sept. 11 for adults and children aged two years and older with spinal muscular atrophy who are already receiving an SMN2-targeted treatment.

Spinal muscular atrophy is a rare genetic disease that causes progressive muscle weakness and loss of motor function. Scholar Rock said Isembyld is the first and only muscle-targeted treatment approved for SMA. However, SRRK shares are down more than 12% since the approval.

Biofrontera’s (BFRI) Ameluz also received an FDA decision ahead of its September 28 PDUFA date. The label expansion covers Ameluz photodynamic therapy for superficial basal cell carcinoma, a common form of skin cancer. BFRI shares have seen an even sharper decline, down 23% since the FDA decision on September 14.

Mirum Gets FOP Drug Approval

On Friday, the FDA approved Mirum Pharmaceuticals’ (MIRM) Atebrioz for patients aged 12 and older with fibrodysplasia ossificans progressiva (FOP), an ultra-rare genetic disease that causes bone to form in muscles and other soft tissues, gradually restricting movement.

Mirum expects Atebrioz to become available in the U.S. in October.

The biotech firm acquired worldwide rights to Atebrioz from Incyte (INCY) earlier this year. Incyte remains eligible for milestone payments and royalties from the drug.

MIRM shares closed Friday’s session 0.4% higher and are currently trading up 3% in pre-market.

How Have BFRI, BMY, SRRK, MIRM And INCY Performed YTD?

Among these stocks, Biofrontera (BFRI) has gained the most this year, rising nearly 63%, followed by Incyte (INCY) at more than 22% and Bristol Myers Squibb (BMY) at about 18%. Scholar Rock (SRRK) and Mirum Pharmaceuticals (MIRM) have each gained roughly 13%.

Also read: Critical Minerals Wrap: CRML, GRML Surge On Greenland Deal, US Stockpile Push Accelerates As Trump-Xi Talks Put Rare Earths In Spotlight

For updates and corrections, email newsroom[at]stocktwits[dot]com