5 FDA Announcements To Pay Attention To In The 2023 Q1

The biotechnology sector had a decline in 2022 that went beyond just falling stock prices. Additionally, only 37 new drugs were approved by the FDA’s primary review office last year, the lowest number since 2016.

Yet there is cause for optimism, as the number of treatments the FDA approves each year might vary. According to RBC Capital Markets analysts, the 2022 approvals of the ALS medication from Amylyx Pharmaceuticals and the Type 1 diabetes medication from Provention Bio demonstrate a growing tolerance for medications with conflicting or limited data. Drug rejections and FDA-ordered trial suspensions decreased from 2021 to 2022. Three gene therapies for genetic disorders were also approved by the regulator, showing that Commissioner Robert Califf looks open to innovation, they stated.

Early in 2023, that belief might be put to the test. The FDA is debating whether to approve a gene treatment for haemophilia A as well as a new Alzheimer’s medication.

Following are 5 choices to keep an eye on in the first quarter of 2023.

Leqembi from Biogen and Eli Lilly’s donanemab for Alzheimer’s disease

Eisai, both of which U.S. regulators provisionally approved in the week ending  January 7, may soon have competition. Donanemab, an Eli Lilly medicine with a similar mechanism of action that may hit the market within the next two months, is closely behind it.

Donanemab’s expedited clearance would allow Lilly to reap some rewards for the many years it has spent funding Alzheimer’s research. But, at least in the short term, the company might have a difficult time gaining market share.

In a sizable Phase 3 experiment, Leqembi has already been demonstrated to modestly reduce the progression of the disease. Comparatively, Donanemab’s application is mostly based on findings from smaller, mid-stage studies. Near the middle of the year, possibly many months after the medicine is made accessible, late-stage data are anticipated. Doctors may have to make difficult choices about which medication to recommend in the interim, and Lilly may need to be cautious when setting a price. When Biogen set the price of its earlier Alzheimer’s medicine, Aduhelm, at $56,000 per year despite conflicting clinical trial results, insurers fiercely resisted. Leqembi’s price was reduced by Eisai to $26,500 per year, raising the threshold.

Leqembi and donanemab, if permitted, are unlikely to be used extensively under Medicare’s stringent coverage guidelines until the FDA issues a full clearance.

Breast cancer drug elacestrant from Menarini Group

The osteoporosis medicine Tymlos was the main product of the biotech company Radius Health before it was bought by two private equity groups last year. Elacestrant, the second agent, is a breast cancer drug that may have a higher chance of finding commercial success.

Elacestrant is one of a class of drugs, referred to as SERDs for short, that are taken orally and block and breakdown hormone receptor proteins. It is hoped that these medications will take the place of fulvestrant, an injectable treatment that is frequently provided to breast cancer patients whose condition is hormone-driven. Their future, however, is uncertain. One crucial trial has only been won by elacestrant. There were two Roche and Sanofi medications that didn’t work.

Menarini Group, the Italian pharmaceutical company that licenced elacestrant from Radius in 2020, could get the first shot at a potentially sizable market if the FDA approves by the February 17 deadline. Menarini is requesting authorization of the medication for those with ER-positive, HER2-negative advanced breast cancer, which is a frequent kind of the disease. For this patient population, elacestrant will be the first marketed endocrine therapy in more than 20 years.

In a December research note, Stifel analysts stated that if the medicine is authorised, uptake in the second-line situation might be significant. They based their statement on conversations with breast cancer specialists. However, it may depend on the amount Menarini decides to charge as well as if the FDA approves it for all patients or just the group that seems to benefit the most.

It’s possible that Menarini won’t have the market to itself for very long. Rival therapies are currently undergoing advanced testing from AstraZeneca, Lilly, and Arvinas. B. Fidler

Hemophilia A medication, Roctavian, from BioMarin Pharmaceutical

It’s possible that BioMarin will release Roctavian, its gene therapy for haemophilia A, on the American market in 2023. However, BioMarin may yet need to overcome another challenge on its already protracted path to FDA clearance.

For haemophilia A, the most prevalent kind of chronic blood illness and a condition brought on by a deficiency in a clotting protein, BioMarin has spent years researching Roctavian. The FDA authorised Hemgenix, a gene therapy for haemophilia B, in November. Like Hemgenix, Roctavian is intended to be a long-term treatment that increases levels of clotting protein, shielding patients from spontaneous bleeding and eliminating the need for further medications. Clinical trials have demonstrated its capacity to do so, but there are also indications that its advantages may diminish over time.

The data provided by BioMarin was sufficient to persuade European regulators, who last August approved Roctavian. The FDA, however, has been more difficult for the company to persuade. In 2020, the agency denied Roctavian and demanded further information on the durability of the treatment’s effects. Following the collection of those data, BioMarin submitted a new application. The FDA is currently analysing the entire three-year data from Roctavian’s major trial, which was announced on January 8. As a result, the business recently hinted that even that assessment may continue past its deadline of March 31.

Analysts are mostly upbeat despite the ongoing uncertainty. According to SVB Securities analysts, the FDA appears to be more confident with haemophilia gene treatments as seen by the FDA’s clearance of Hemgenix in December. Additionally, RBC analysts pointed out in December that the agency had changed its mind on holding an advisory committee meeting, eliminating a potential roadblock.

If approved, BioMarin will maintain its lead over Pfizer and Roche, which are also working on haemophilia A gene treatments. 

Trofinetide from Acadia Pharmaceuticals treats Rett syndrome

Acadia has now failed twice in its attempts to get the FDA to expand the approval of a medicine for Parkinson’s disease that it markets under the name Nuplazid. The regulator denied the company’s requests to use Nuplazid longer to treat psychosis associated with dementia in August of last year and again in April of this year.

Acadia’s regulatory issues have hurt the business; since the first indications of difficulty with the first application, shares have dropped by over two-thirds.

With its medicine, trofinetide, which it has been researching for the neurological condition known as Rett syndrome, the business is hoping for a different result. The FDA is currently reviewing the application, and March 12 is the deadline for a decision.

When compared to a placebo in clinical trials, treatment with trofinetide for girls and young women with Rett syndrome resulted in higher scores on two assessments of the condition’s symptoms. Trofinetide would provide patients with a much-needed choice if it were approved and would be the first treatment for Rett. Success would also be advantageous for Acadia, possibly offsetting the company’s difficulties in marketing Nuplazid in its authorised prescription for Parkinson’s disease. Additionally, a unique regulatory voucher that can be sold may be included with the approval.

In a contract signed with Neuren Pharmaceuticals in 2018, Acadia obtained the rights to trofinetide in North America. Neuren would be owed one-third of the drug’s worth in the event that the FDA approves it and gives a voucher. 

Trodelvy for breast cancer from Gilead Sciences

The HIV and hepatitis C medications Gilead is most known for are two extremely successful industries that were sparked via acquisitions.

With the $21 billion acquisition of Immunomedics two or more years ago—the largest acquisition in the history of the company—the California biotech aims for a similar level of success. The acquisition of Trodelvy, a cancer medicine, is seen by Gilead as the key to its push towards oncology, a field of study that it has traditionally shunned.

Analysts, though, aren’t yet persuaded that the transaction was worthwhile given its hefty cost. The difficult-to-treat “triple-negative” kind of breast cancer is presently approved for Trodelvy. Its chances might be improved by expanding its application to the most prevalent type of HR-positive and HER2-negative breast cancer. Initial findings from a significant clinical trial were encouraging, however they were seen as providing only a slight benefit. Follow-up statistics showed that the therapy increased survival by three months.

Todelvy’s approval for usage in this situation, for adults with selected patients metastatic or locally advanced breast cancer that cannot be removed surgically, is now being reviewed by the FDA. A choice must be made by February.

Even if a clearance is given, Enhertu, another breast cancer medication, will be a fierce competitor for Gilead. Enhertu, a drug created by AstraZeneca and Daiichi Sankyo, was lately approved by the FDA for use in treating malignancies that are thought to be “HER2 low.” It has demonstrated promising outcomes in testing.

Analysts now predict that Trodelvy would be used in this type of breast cancer after Enhertu.